[HN Gopher] Gene therapy restores hearing to children with inher...
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       Gene therapy restores hearing to children with inherited deafness
        
       Author : keploy
       Score  : 478 points
       Date   : 2024-06-08 20:40 UTC (1 days ago)
        
 (HTM) web link (cosmosmagazine.com)
 (TXT) w3m dump (cosmosmagazine.com)
        
       | gota wrote:
       | This stuff seems miraculous.
       | 
       | > "Through minimally invasive surgery, Shu injected adeno-
       | associated virus (AAV) engineered to carry and deliver
       | functioning copies of the human OTOF transgene, into the
       | children's inner ears. "
       | 
       | Can't read that and not get curious, excited, for the future.
       | 
       | Congratulations to all those involved in the research and thanks
       | to all who contributed to it - including all taxpayers if public
       | funding was given, even indirectly (i.e. in the schooling and
       | education, grants, for said researchers)
        
         | dyauspitr wrote:
         | I can't wait for a geneCAD where we for example, design wings
         | for a human body and then it works out the rest and generates a
         | CRISPR based AAV that you can inject yourself with. True race
         | and sex changes too.
         | 
         | Hopefully in the next 50 years but that's wishful thinking.
        
           | kevin_thibedeau wrote:
           | There are potential serious downsides if these inserted genes
           | end up on a different chromosome than normal and become
           | carried by gametes to offspring who may suffer from
           | overexpression.
        
             | dyauspitr wrote:
             | I presume at the point we can generate CAD like designer
             | gene modifications, overexpression would just be another
             | problem that can be fixed by more modification.
        
               | AuryGlenz wrote:
               | Eh, maybe you'll run into the problem the Asgard had in
               | Stargate. Too much editing over time = couldn't procreate
               | anymore. Little bugs add up.
        
               | ben_w wrote:
               | That was a plot device that didn't make sense to me: they
               | could upload and download a functional copy of their
               | brains (and also human brains), but they didn't keep a
               | backup copy of their old DNA?
        
           | antegamisou wrote:
           | We need to keep away biotech OpenAI-wannabes apparently.
        
           | mylons wrote:
           | it's happening! i just finished something that assists in
           | "CRISPR Design" and helps gene editors.
        
           | nkrisc wrote:
           | Taking it seriously for a moment, that still seems outlandish
           | even in a sci-fi scenario. The amount of changes that would
           | be necessary to the body for a human to achieve autonomous
           | powered flight would be substantial.
           | 
           | Given our overall density, we would need enormous wings and
           | musculature. Consider that the heaviest flying birds alive
           | today weigh maybe 40lbs and have a wingspan of 8ft or so. If
           | we naively extrapolate that linearly then an adult weighing
           | 160lbs would need a 32ft wingspan.
           | 
           | There were flying pterosaurs that weighed much more than the
           | heaviest flying birds today, but they were also the size of
           | small planes and it's a bit of a debate how well they even
           | flew. They may have struggled to even take flight.
           | 
           | So injecting yourself with some designer DNA isn't just going
           | to make you grow wings, it would have to completely transform
           | you into a completely different creature, simply due to the
           | physics of flight.
           | 
           | Additionally, you need not only to be able to fly, but your
           | body needs to function well enough to stay in this new state.
           | You would also likely need an enormous caloric intake to
           | support the massive new growth, or it would take a very, very
           | long time.
           | 
           | It wouldn't be designing wings for a human, it would be
           | designing a be creature _based_ on a human but not human at
           | all, really. Call me shortsighted but I don't really see how
           | that will ever be possible.
        
             | dyauspitr wrote:
             | Your point seems to be that massive changes are required to
             | achieve wings. I'm saying that's not impossible and an
             | acceptable modification to whoever wants to do this to
             | themselves. As long as you can maintain the brain as is, I
             | don't see there being a problem with radically modifying
             | the rest of the body.
             | 
             | Argentavis magnificens had a wingspan of around 24ft and
             | weighed around 72 kgs or a 160lbs which is close to what an
             | average human weighs.
             | 
             | If you compare growth rates to other mammals like
             | elephants, I doubt a change like that would take much more
             | than 5-10 years.
        
               | nkrisc wrote:
               | > As long as you can maintain the brain as is,
               | 
               | That's the question, can a human sized body that can fly
               | additionally support a human sized brain? I suspect you
               | would have to spend nearly every waking minute eating to
               | support both. Or at least a substantial portion of your
               | day. No time for hacking, gotta eat.
               | 
               | You'll probably have to pay full up front for this, as no
               | one will finance you since you'll be too busy foraging
               | for sugary fruit or carrying livestock off to your eyrie
               | to work a job to pay it back.
               | 
               | Finally, this raises another question I won't even
               | attempt to answer: is it even possible for you to still
               | be "you" in another body? How responsible is your body,
               | beyond just your brain, responsible for making you who
               | you are?
        
               | nonameiguess wrote:
               | It's about as close to impossible as you can get. If you
               | tried to do it in the womb, too much deviation from a
               | regular human fetus won't be carried to term. If you try
               | to do it after growth plates have closed, you'd have to
               | destroy every bone in the body first, which would kill
               | you. That gives you some kind of post-birth, pre-puberty
               | window over which some sufficient level of body
               | remodeling can at least happen in principle, but you seem
               | to be underestimating the level of remodeling to do this.
               | You'd need to drastically reduce bone density, which
               | would leave you extremely susceptible to injury. You'd
               | have to undo human adaptations in the spine and pelvis
               | for upright posture, which would be extremely painful.
               | You'd need to effectively swap out the glutes with pecs
               | and undo the adaptations for brachiating arms. Things
               | like your eyes and ears and basic breathing apparatus are
               | not well-adapted for flight. Things like where blood and
               | lymph and other bodily fluids tend to pool in the human
               | body versus where they do in the bodies of flying
               | animals. Just as laying down for too much time will cause
               | it to pool in places your body can't easily clear right
               | now, being horizontal for flight would have the same
               | effect.
               | 
               | It's not just a matter of growing wings. And yeah, the
               | energy demands of making all these changes, as others
               | have pointed out. The only kinds of animals that go
               | through this level of non-fetal metamorphosis are insects
               | with weights measured in the tens of grams, energy needs
               | that kind be sustained by something like a cocoon. How
               | would you meet the energy demands of an adult human going
               | through metamorphosis? You couldn't do it by eating, not
               | only because your gut can't actually digest the amount of
               | food you'd need (you don't have an elephant gut) but also
               | simply because being conscious through the process,
               | unlike insects in a cocoon, would be so absurdly painful
               | that I doubt you'd be able to function and do anything at
               | all, let alone spend all of your time finding and eating
               | food. You'd need to be put into a medical coma and
               | injected with intravenous nutrients to have any shot at
               | all.
               | 
               | Why on earth would we ever try this? In reality, giving
               | yourself genes to grow wings would just kill you.
               | 
               | Look at this picture from Wikipedia showing Argentavis
               | side by side with a human: https://en.wikipedia.org/wiki/
               | Argentavis#/media/File:62628-A...
               | 
               | What sorts of processes do you imagine are necessary to
               | stretch the human body to the size of the Argentavis body
               | without adding any weight? It'd be like getting flattened
               | by a steam roller, then drawn and quartered. Bodily
               | tissue isn't balloons.
        
             | slfnflctd wrote:
             | "...are you suggesting coconuts migrate?"
             | 
             | Seriously, lesser-schooled folks don't realize the extent
             | to which evolution took tiny variations over an extremely
             | long time to achieve physical flight. The way it arose
             | differently in pterosaurs, birds and bats is fascinating.
             | 
             | I think younger children are capable of understanding more
             | about genetics than we often suppose. More of these
             | concepts should be taught at younger ages.
        
               | dyauspitr wrote:
               | I have a bachelors in bioengineering, I'm not a "lesser
               | schooled individual". Just because evolution took a long
               | time doesn't mean we have to, you can run millions of
               | simulations virtually, something that nature has to do
               | over millions of years. Are the simulations going to be
               | perfect? Probably not but there's a lot that can be
               | gleaned through simulations combined with observations in
               | the real world.
        
             | awwaiid wrote:
             | (a) I like your analysis (b) maybe the wings are just for
             | show so that you flap them out at clubs every now and then
             | right when the music hits the drop or something and
             | everyone is like WHHAAA????!!!!
        
             | JumpCrisscross wrote:
             | > _it would have to completely transform you into a
             | completely different creature, simply due to the physics of
             | flight_
             | 
             | In OP's defence, they never mentioned flight. Maybe they
             | just want a peacock train.
        
               | nkrisc wrote:
               | Ok, fair enough. I hadn't considered that. In my mind,
               | wings you can't fly with are worse than no wings.
        
               | galangalalgol wrote:
               | Tell that to am emu or ostrich, or don't, their claws
               | aren't vestigial.
               | 
               | I would opt for smaller tweaks like an extra rhodopsin
               | folding with longer features so I could see near (like tv
               | remotes) ir. re-enable hibernation. Things some mammals
               | already have would be good starts.
        
               | nkrisc wrote:
               | I meant as a human.
        
             | diego_sandoval wrote:
             | All that energy will be better spent designing jetpacks
             | powered by portable nuclear reactors.
        
             | hinkley wrote:
             | A futurist I knew of long ago pointed out that the Moon's
             | gravity is light enough that the human skeleton could
             | achieve sufficient lift without sustaining damage in the
             | process.
             | 
             | True, you might have to be a junior Olympian to do it, but
             | it's possible.
             | 
             | On earth your pectorals would tear off of your sternum long
             | before you achieved takeoff.
        
               | BurningFrog wrote:
               | I thought the same thing.
               | 
               | The lack of Moon atmosphere is a real problem though.
        
               | abecedarius wrote:
               | Heinlein, "The Menace from Earth"
        
               | hinkley wrote:
               | Don't bump into the top of the dome. You'll get charged
               | for disinfecting the equipment.
        
           | taf2 wrote:
           | That would be cool - maybe a simple 20/20 vision would be a
           | great next step after hearing
        
           | akoboldfrying wrote:
           | Let's try to fix the world's vast number of actual, boring
           | health problems before trying to add the equivalent of
           | spoilers and chrome-plated hubcaps to perfectly healthy human
           | bodies.
        
             | dyauspitr wrote:
             | You can do both things at the same time.
        
               | akoboldfrying wrote:
               | That's true if we assume unlimited resources and equal
               | awareness/perceived social value of the two applications.
               | But I think the former doesn't hold, and the latter is
               | greatly influenced by sites like HN, where I'm
               | disappointed to consistently (if not exclusively) see
               | breathless proposals for ways to design shinier hubcaps
               | whenever some promising new technology appears.
               | 
               | Yes, they can and likely will both happen to some extent,
               | but I think they aren't independent, so I feel justified
               | in trying to nudge the public conversation back towards
               | the issues that I think matter more.
        
               | evilduck wrote:
               | Cosmetic surgery for vanity has helped improve the
               | techniques and procedures and even the number of skilled
               | practitioners that can then help those with
               | disfigurements and deformities.
        
               | JumpCrisscross wrote:
               | Hell, cancer treatment for pets has advanced human
               | oncology [1]. Scientific discovery is rarely zero sum.
               | 
               | [1] https://www.upstate.edu/whatsup/2019/0220-treatment-
               | for-cani...
        
               | _factor wrote:
               | And cigarettes have benefited the lung cancer research
               | centers. Just because it works doesn't make it the best
               | course.
        
               | JumpCrisscross wrote:
               | > _breathless proposals for ways to design shinier
               | hubcaps whenever some promising new technology appears_
               | 
               | I wouldn't underestimate the emotional toil of dealing
               | with illness and death [1].
               | 
               | Tackling these problems head on requires (a) exposing
               | researchers to that toil and (b) removing from the pool
               | anyone who doesn't want to do that. Given how much of
               | Silicon Valley culture is built on borderline-ludicrous
               | optimism (once it's over the border it no longer
               | qualifies as building), it makes sense that the indirect
               | approach finds resonance here in a way the direct one
               | does not.
               | 
               | Where your argument finds ample purchase is in the
               | asymmetry of idiot luxury spending in our society to
               | basic and applied research of any kinds, wings or Wilm's
               | tumour.
               | 
               | [1] https://www.statnews.com/2016/09/19/mental-health-
               | doctor-res...
        
               | smegger001 wrote:
               | In all likelihood if we were to get to the point of it
               | being safe and cheap enough for genetic engineering
               | cosmetic "upgrades" then that would through economies of
               | scale drive enough money that medical research funding
               | would be a solved problem. Also anyone wanting the chrome
               | pipes and spoilers package is going to want their engine
               | tuned. Whats the point of having functional wings if you
               | heart blows out trying to use them.
        
               | inglor_cz wrote:
               | "they aren't independent"
               | 
               | That interdependence may be beneficial, though.
               | 
               | Experience and revenues from cosmetic treatments will
               | help health-restoring treatments.
        
               | jdietrich wrote:
               | Economies of scale. Shiny hubcap manufacturers will very
               | generously subsidise the R&D for other, more worthy users
               | of a technology. They aren't competitive or independent,
               | they're synergistic. The billions of dollars we "wasted"
               | on making video games look more realistic gave us a
               | revolution in high-performance computing that nobody
               | could have created intentionally.
        
               | refurb wrote:
               | Each gene therapy is a unique "medicine". There is little
               | economy of scale, at least for the hardest part.
        
               | ninjanomnom wrote:
               | That's due to a lack of theory and useful abstractions in
               | the tools used to modify genes. We're so early on in the
               | development of that field that if this were computers,
               | we'd still be assembling devices by arranging logic gates
               | by hand. Custom cosmetic gene therapy is a great way to
               | incentivize the better tools.
        
               | UniverseHacker wrote:
               | Moreover, they are at this stage the exact same problem-
               | requiring not competing but identical research: we need
               | to understand generally how biology works enough to
               | predictably engineer it.
        
               | JumpCrisscross wrote:
               | > _they are at this stage the exact same problem-
               | requiring not competing but identical research_
               | 
               | I'd go one step further in arguing they're complementary.
               | The personalities that will work on _e.g._ wings or
               | longevity are not the types drawn to curing diseases,
               | much less the boring ones.
               | 
               | Broadening the field from solving mundane problems to
               | solving daring ones is net positive. You gain
               | personalities that would have otherwise stayed away. (You
               | see something similar in space programmes.)
        
               | abecedarius wrote:
               | And a huge part of what steered talented people into
               | programming over biomedicine was the relative freedom.
               | The more you can do without the drag of convincing a vast
               | bureaucratic/political machine to let you try your idea,
               | the more people will contribute to progress.
        
               | refurb wrote:
               | Actually you can't because there is a limit to resources
               | in both time and money.
        
             | LoganDark wrote:
             | I wish it were possible to achieve an entirely _non-human_
             | body, but I don 't think that's going to happen within any
             | of our lifetimes.
        
               | telesilla wrote:
               | Make sure to read Daniel Dennett before you change too
               | much, you might forget who you are and become someone
               | else entirely.
               | 
               | https://en.wikipedia.org/wiki/What_Is_It_Like_to_Be_a_Bat
        
               | LoganDark wrote:
               | I have Dissociative Identity Disorder, so I already
               | become someone else all the time...
               | 
               | In all seriousness, at times I do genuinely wish species
               | transition were possible. Imagine gender dysphoria but
               | for species, so species dysphoria. It may sound insane,
               | but honestly so can the entire concept of dissociative
               | identities.
               | 
               | I don't know if I'd want to change my brain, but the
               | physical properties of the body definitely. I want the
               | body to be a fluffy quadruped...
               | 
               | Thank you for the link though, that is very interesting.
               | It is intuitive, but not something that normally comes to
               | mind~
        
               | ben_w wrote:
               | I remember the Therian and Were community, back in the
               | day. Some overlap with the furry community, but not a
               | perfect subset.
        
               | simianparrot wrote:
               | It is the very definition of insane. That doesn't mean we
               | shouldn't have sympathy for it, but it's important to
               | recognise when certain aspects of our psyche isn't sane
               | or good and accept that.
        
               | abdulhaq wrote:
               | you are what you are, there's no escape from that
        
               | vsuperpower2020 wrote:
               | I hope you're joking about wanting to become a "fluffy
               | quadruped". Or, if not, I see why we had asylums.
        
             | Teever wrote:
             | What do you get from admonishing someone for saying the
             | kinds of things that they just said?
        
               | akoboldfrying wrote:
               | Why do you consider an attempt to focus a public
               | discussion on the most important uses of a new technology
               | to be an admonishment?
        
               | fragmede wrote:
               | it certainly reads as one, and calling them "most
               | important" reveals your bias that those uses are more
               | important too.
               | 
               | Like it or not, the way society is currently arranged,
               | making stuff for rich people is more profitable than
               | making things for poor people, and greed can kick in, so
               | people do things out of love of money rather than
               | necessity. were the deck stacked more equal, maybe things
               | would be different, but human psychology is devilishly
               | complex.
        
             | marvin wrote:
             | Just ensure that there's good a insurance system to finance
             | the rare health issues, and the market will take care of
             | the hubcaps and spoilers.
             | 
             | Solving e.g. male pattern baldness or bone loss from
             | peridontitis means guaranteed billionaire from out-of-
             | pocket treatments alone.
        
             | gurumeditations wrote:
             | Sex changes/gender transitions is an actual boring health
             | problem. The inability to assume the correct gender for a
             | trans person often leads to social rejection, violence,
             | poverty, prostitution, murder, and suicide.
        
               | Almondsetat wrote:
               | If through gene editing you could also erase someone's
               | disphoria what option would you choose?
        
               | wouldbecouldbe wrote:
               | That's like trying to cure depression by getting rich. It
               | hardly ever works
        
               | Almondsetat wrote:
               | What?
        
               | wouldbecouldbe wrote:
               | Cutting off body parts to treat body dysphoria is
               | something that should be done with great care and rarely
               | leads to solving the underlying mental problems.
               | 
               | Trans is a much discussed variant that is hard to have a
               | normal talk about, but there are many more variants, for
               | instance women hating their breast & wanting to remove
               | them, I think most people here would agree that intensive
               | psychological treatment are preferable to actually
               | removing healthy body parts. But in the end an adult can
               | do what they want.
        
               | sillysaurusx wrote:
               | Isn't it obviously good to get rid of someone's problem?
               | Otherwise it wouldn't be a problem.
        
               | Almondsetat wrote:
               | If your perception doesn't match your body, would you
               | prefer to change the perception or change the body?
        
               | sillysaurusx wrote:
               | It seems pretty clear to me that changing one's body is
               | preferable to changing who you are. The former is
               | something you're born with, but the latter is something
               | you can decide for yourself.
        
               | Almondsetat wrote:
               | Suppose, as you do, that your body is not who you are.
               | Then your identity lies solely in your brain. But if
               | that's the case why would disphoria even exist?
               | 
               | If you feel like a man but you don't look like a man, why
               | could that cause any identity problems? It must mean your
               | body is actually part of your identity, so changing it
               | changes who you are
        
               | sillysaurusx wrote:
               | The problem with this argument is that it leads to the
               | conclusion that all change is pointless. If your body is
               | who you are, and you can't control it, then it's best
               | just to accept whatever you're given.
               | 
               | That's contrary to most of human history, where we
               | specifically try not to take what we're dealt.
               | 
               | It's useful to ask yourself: why should vaccines be
               | "allowed" (or "accepted" or "they're good") but body
               | change shouldn't be? They're both as artificial as a
               | Twinkie.
        
               | groovy2shoes wrote:
               | Erasing someone's dysphoria would also fundamentally
               | change their identity and personality. If I were to
               | suffer a head injury today that so altered my
               | personality, the result would be very unpredictable. I
               | could become a better person, or a worse one. I could
               | lose my wife, my career, everything. But even if the
               | outcome were positive, I wouldn't be _me_ anymore. This
               | me would cease to exist, replaced by a new one.
               | 
               | On the other hand, a bodily alteration is much more
               | predictable, and, importantly, I'd still be _me_. I
               | wouldn 't become some other person.
               | 
               | This me has an instinct for self-preservation. Thus, if
               | both options were available, I would absolutely choose
               | transition over erasure.
        
               | Almondsetat wrote:
               | You seem to come from the point of view that body and
               | mind are quite separate, and that changing your body
               | through surgery and hormones and other chemicals has less
               | effects on the "real you" than a change in brain wiring
               | would cause.
        
             | jmcgough wrote:
             | Most of those problems in the US are caused by lifestyle,
             | and can't really be changed without behavioral
             | modification. And a lot of our health issues are a matter
             | of poor access to healthcare.
        
               | landryraccoon wrote:
               | This assumes that the fundamental nature of human beings
               | can't be changed, which is the presupposition that
               | genetic engineering denies.
               | 
               | The entire point of genetic engineering is to try to
               | engineer people at the cellular level. A fanciful example
               | would be modifying human metabolism to be more similar to
               | those of birds that consume most of their calories in the
               | form of simple sugars. Humans can't eat a diet of 100%
               | sugar and remain healthy, but other animals can. It may
               | be possible to change that fact, if we know how to edit
               | our genes.
        
               | xedrac wrote:
               | Are you saying you could see sugar being somehow folded
               | into the many different proteins that make up the vast
               | number of tissues in our bodies. Or are you saying our
               | tissues could be constructed out of sugar crystals
               | instead?
        
             | Almondsetat wrote:
             | Has there been any cases of this happening for other
             | technologies?
             | 
             | That's like saying "now that we invented canals let's try
             | to fix the world's irrigation problems before making
             | fountains and water parks"
        
             | BurningFrog wrote:
             | We're 8000 million people.
             | 
             | We can, and always will, work on many different things at
             | once.
        
               | vsuperpower2020 wrote:
               | The amount of people who can actually work on anything
               | meaningful in this field is double-digit at best. Sure,
               | if you add up "many different things" you have plenty of
               | people who can work on plumbing, programming, and gene
               | editing. What are you going to do for geneCAD? Right,
               | absolutely nothing. It doesn't matter how many of you
               | there are; you could be 8000 million or 8000 trillion
               | people and still accomplish nothing.
        
               | gibolt wrote:
               | This assumes things are static. There are millions of
               | programmers now, but certainly weren't 100 years ago.
               | 
               | The tools to investigate this will get cheaper and
               | easier, driving more people into the field hoping for the
               | next big win, a big payout, or just to make an impact.
               | 
               | Anyone can make a small circuit design have have a fab
               | print it for you. The same could happen here, all you
               | need to do is provide the sequence
        
             | m3kw9 wrote:
             | Na, this isn't it, you one of them that talks let's solve
             | poverty before spending money on xyz
        
           | narrator wrote:
           | No doubt you'll see an Android/Apple split and the Apple
           | people won't be able to date the Android people because of
           | incompatible augmented pheromones that smell funny if you're
           | not on the same platform.
        
           | UniverseHacker wrote:
           | Synthetic biology CAD software does exist, and works better
           | than most people would expect, but of course are not at the
           | level you are imagining yet.
        
             | Nesco wrote:
             | Could you provide some resources/links please?
        
               | UniverseHacker wrote:
               | It's hard to summarize because there are about a dozen
               | different categories of things that could reasonably
               | considered "biological CAD" and often would all be used
               | together in a single synthetic biology project.
               | 
               | For example: Retrobiosynthesis simulates biochemistry
               | backwards, to find the steps necessary for a biological
               | system to build something, usually a small molecule.
               | Galaxy-SynBioCAD / Retropath would be one example:
               | https://jfaulon.com/galaxy-synbiocad-portal/
               | 
               | Constraint based metabolic modeling models cellular
               | metabolism, and lets you simulate adding and removing
               | chemical reactions to a cell, and predict the outcomes.
               | COBRApy would be one example software tool:
               | https://opencobra.github.io/cobrapy/
               | 
               | Design editors for DNA plasmids, like the Teselagen
               | design editor let you construct DNA sequences
               | representing new biological capabilities to be added to
               | an engineered cell, which can then be synthesized or
               | constructed. Teselagen design module:
               | https://teselagen.com/design-module/
               | 
               | Generative AI systems can 'hallucinate' functional
               | proteins and DNA sequences that meet a design
               | specification for function and/or shape. For example,
               | GenerateBio's Chroma model can literally take a 3D file
               | designed in a standard CAD program, and then
               | automatically come up with an amino acid sequence that
               | will fold into a protein with that exact 3D shape- and it
               | actually works. https://github.com/generatebio/chroma
               | 
               | An emerging field is coupling all of these types of tools
               | with predictive models to enable 'inverse design' where
               | you create a spec of what you want, such as a material
               | with some desired properties, and it will automatically
               | suggest biological routes to it.
        
           | inglor_cz wrote:
           | Our bodies are very much not suitable for flight. Birds look
           | differently from the inside.
           | 
           | It might prove easier to grow biological pylons for external
           | jet engines...
        
           | hinkley wrote:
           | Dan Simmons, Hyperion Cantos. "Biosculpting"
        
           | TyrianPurple wrote:
           | Excuse me, are you The Qu?
        
         | m3kw9 wrote:
         | I thought you can't just inject genes and then the body will
         | start replicate it. Does it need periodic injections?
        
           | rguiscard wrote:
           | That is why the gene is put into a harmless virus which will
           | injects the gene into cells. It takes advantage of what virus
           | usually does.
        
           | toufka wrote:
           | If the DNA is in the cell's nucleus, the DNA will be utilized
           | to produce whatever gene is encoded in the DNA. But if the
           | DNA is just floating around on its own it will not survive a
           | cell division event and the material would need to be
           | periodically reinjected to keep working. However, if the
           | injected DNA is part of a full chromosome, it will be
           | replicated when the cell divides - and will be permanent as
           | long as the cell or its progeny survive.
           | 
           | Some viruses will just inject the DNA into cells, but will
           | not become part of the cell's genome ("transient"
           | transduction). Other viruses (like lentiviruses and these
           | adeno-associated viruses [AAVs]) inject their DNA not just
           | into the cells, but also have machinery that splices their
           | payload DNA directly into the cell's chromosomes
           | ("integrated"). The location in the genome of the splicing
           | event is relatively random. Random is not necessarily great
           | as it could interrupt other genes already in the chromosome.
           | CRISPR is a now-famous tool that helps "integrate" DNA into a
           | specific spot in the genome by being guided to a specific
           | location with a small piece of a specific sequence.
           | 
           | Once the DNA is integrated, any cell, and any of the cell's
           | progeny, will produce or "express" the gene on the delivered
           | DNA. In this case, they delivered the 5991 characters of DNA
           | associated with the OROF gene [1].
           | 
           | [1] https://en.wikipedia.org/wiki/Otoferlin
        
             | rekttrader wrote:
             | Thank you for this description of how it works. It seems
             | like truly beautiful and terrifying magic.
        
             | bglazer wrote:
             | This is actually not totally correct. AAV's form episomes,
             | which are circular DNA structures that are basically extra
             | chromosomes. The fact that AAV's don't randomly insert
             | themselves into DNA makes them safer for clinical uses like
             | this.
             | 
             | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2519600/
        
         | tim333 wrote:
         | I'm sure there will be other treatments based on AAV and
         | CRISPR. I'm interested in ALS as a relative has it and that
         | stuff is one of the only approaches that might work. The had a
         | go on mice with so so results - lived longer but no cure
         | https://www.cell.com/molecular-therapy-family/molecular-ther...
        
       | Something1234 wrote:
       | Wait until they have kids. The deafness gene will be passed
       | along. Soon enough we'll be like the cars with the hardware
       | without the software or the locked features.
        
         | david38 wrote:
         | We have this already. As a species we are susceptible to many
         | diseases which we have to take vaccines for, every generation.
        
           | pfannkuchen wrote:
           | I don't think susceptibility to viral diseases like measles
           | is typically regarded as a genetic defect, is it? Any more
           | than being flammable is a genetic defect, anyway.
        
             | roywiggins wrote:
             | There are a few people who are genetically immune to HIV,
             | but it's quite rare. Whether everyone else is therefore
             | genetically deficient is sort of a matter of perspective, I
             | suppose.
             | 
             | https://en.m.wikipedia.org/wiki/Innate_resistance_to_HIV
        
         | bitcurious wrote:
         | Fortunately won't be a big issue, as:
         | 
         | >OTOF-related deafness is inherited in an autosomal recessive
         | manner. If both parents are known to be carriers, each sib of
         | an individual with OTOF-related deafness has at conception a
         | 25% chance of being deaf, a 50% chance of having normal hearing
         | and being a carrier, and a 25% chance of having normal hearing
         | and not being a carrier.
         | 
         | source: https://www.ncbi.nlm.nih.gov/books/NBK1251/
        
         | patmorgan23 wrote:
         | How is this any different from people who suffer from other
         | genetic disorders that we have treatments for? Or people who
         | just suffer from a chronic disease that requires regular
         | treatments/medication for them to stay alive/health? (HIV,
         | Diabetes, etc)
        
         | BriggyDwiggs42 wrote:
         | Deaf people can already have kids
        
         | tithe wrote:
         | And we'll cure them too.
        
         | dfadsadsf wrote:
         | In vitro fertilization (IVF) combined with preimplantation
         | genetic screening (PGS) is becoming increasingly mainstream.
         | This advanced technique allows us to select embryos with
         | healthy genes, thereby ensuring that babies are born healthy or
         | at least reducing the risk of genetic diseases. Very expensive
         | in US but is getting very affordable in Eastern Europe and
         | Asia.
         | 
         | Another alternative is selective abortions after early genetic
         | testing (though ethic and morale concerns here are huge).
        
           | houseplant wrote:
           | that crazy pro-natalist fascist couple that's been all over
           | the news lately do this. They pick and choose which of their
           | embryos are the most fit overall, and/or which have blond
           | hair I'm sure, and plan to have as many of them as possible.
           | Seems a shame to go through all that, only to beat your kids
           | in front of a newspaper reporter and give them neuroses
           | that'll likely make them rebel against you, but I'm sure if
           | they can invest in crypto stocks they must be very good at
           | predicting the future right?
        
         | teaearlgraycold wrote:
         | Damn. Might as well just kill ourselves because our children
         | inherit our problems. My parents need glasses, I'll need
         | glasses. Fuck.
         | 
         | /s
        
         | 8f2ab37a-ed6c wrote:
         | Any different from people needing glasses?
        
         | zmmmmm wrote:
         | Apart from the other points people have made, it's worth noting
         | that these days parents can avoid having children with
         | inherited genetic disorders through IVF. While there will
         | always be a component of spontaneous mutations resulting in
         | genetic disease, we may approach within a couple of generations
         | a state where most inherited disorders actually disappear or
         | reduce to vastly lower prevalence over time.
        
           | nicolas_t wrote:
           | Some backward countries (like my own France) forbid that due
           | to concern over eugenics.
        
         | jubalfh wrote:
         | ah, an eugenicist. and yet, if i stated that eugenicists should
         | be eliminated from the gene pool, you'd be outraged.
        
           | lazyasciiart wrote:
           | Obviously: for a start, since there's no genetic test for
           | eugenicism, you'd have to wait and eliminate them as adults.
           | They may have already reproduced by then, but there's also no
           | clear mechanism for inheritance so we don't even know if that
           | is a problem. If they adopted children do we consider those
           | kids to have inherited it?
        
           | ThrowawayTestr wrote:
           | The fear of being beholden to a company for your health is
           | not one to dismiss so easily.
        
             | jubalfh wrote:
             | get a better healthcare system.
        
         | nkrisc wrote:
         | I'm sure you have some undesirable genes too, whether you're
         | aware of it or not.
        
         | grantsh wrote:
         | Typical engineers, tipping into eugenics to treat society as an
         | optimization problem...
        
       | roygbiv2 wrote:
       | Gene therapy is coming on well. Hopefully it'll help my kid when
       | he's older. From what I'm being told, the gene that effects him
       | is very large and difficult to fit into the viruses they use for
       | gene therapy. My understanding is very limited so I could be very
       | wrong here.
        
       | speedylight wrote:
       | How hard is it to introduce permanent mutations to DNA in adults,
       | across every cell in the body? That seems like the holy grail of
       | gene editing to me!
        
         | zac256 wrote:
         | This is an area of active research, and there are many issues
         | to solve. A technology that distributes a gene editor
         | throughout the body is called a delivery vector, and it is
         | typically a modified virus.
         | 
         | After the patient is two years old, many large molecules do not
         | pass through the blood brain barrier. Typically most of the
         | treatment ends up in the liver. Viruses and the gene editor can
         | be toxic. Combining the two previous points the correct dose
         | for most of the body can be highly toxic to the liver. The
         | delivery vector may support genes up to a specific size (in
         | kilobases) which may be smaller than the gene you are trying to
         | fit. Sometimes you don't want to target organs that the
         | delivery vector hits. Sometimes you want to target organs that
         | the delivery vector misses. And more.
         | 
         | There is rapid progress being made here and all kinds of
         | caveats and nuance to the above.
        
         | inciampati wrote:
         | How perfect can your reaction be? Can you get to
         | 99.999999999999%?
        
       | sircastor wrote:
       | I'm curious about the effect on the deaf community. I understand
       | there's resistance to "fixing" deafness as some consider it a
       | component of their culture and community, not unlike queer people
       | and the attempts to "fix" them.
       | 
       | https://en.wikipedia.org/wiki/Cochlear_implant#Criticism_and...
        
         | devilbunny wrote:
         | There is a fascinating documentary about this, but I cannot
         | recall its name. One segment talks about a form of sign that
         | represents sound, so that the child learns their family's
         | language (rather than sign language, which is another language
         | entirely). It is very unpopular in the deaf community despite
         | its advantages for daily life.
        
           | lazyasciiart wrote:
           | Sounds like Signed Exact English, which is disliked both for
           | the context around it and for being more awkward than a
           | natural sign language (e.g I think it doesn't take advantage
           | of using positions in space for grammar, it only uses the
           | hand shapes and movements?)
           | 
           | I don't know of any language agnostic phonetic sign used as a
           | first language for children: that sounds about as hard as
           | making them learn through writing instead of talking.
        
             | gugagore wrote:
             | They are likely talking about "cued speech".
        
         | akoboldfrying wrote:
         | >It is important to consider the sociocultural context,
         | particularly in regards to the deaf community, which has its
         | own unique language and culture
         | 
         | Frankly, I disagree. I think a deaf person should be entitled
         | to decline a low-risk treatment that would cure or mitigate
         | _their own_ deafness -- but to make that decision for a child
         | is altogether different. Deliberately restricting a human being
         | 's sense experience throughout their lives should be considered
         | child abuse, in the same basket as female genital mutilation.
         | It's also about the most selfish thing I can imagine --
         | especially given that there's no reason I can see why a hearing
         | child of deaf parents can't grow up "bilingual", learning both
         | spoken and sign languages.
        
           | brenns10 wrote:
           | This presupposes an awful lot. If it were truly a risk free
           | magic wand you could wave, then I could see your point.
           | 
           | But none of the current options are risk free, and I doubt
           | this one will be. They all have risks, side effects, and
           | probably the chance of failure. And then the question is how
           | low of a risk is acceptable to restore a child's hearing.
           | These are people who would live an otherwise healthy and
           | normal life, and they'd grow up in an accepting deaf family
           | which is likely already plugged into a community. I can't
           | imagine how it would feel to have a serious side effect (or
           | worse) impact your child when you know they could have gone
           | without that procedure and lived a happy, fulfilling life.
           | But I also understand the desire to give your child every
           | possible opportunity.
           | 
           | All of that is to say, I think it is reductive to compare
           | this to child abuse or FGM. There's no right decision, and
           | parents absolutely will need to make a difficult choice,
           | hopefully prioritizing their child's safety and future above
           | all else.
        
             | josephcsible wrote:
             | It's often not about the risk, though. It's sadly common
             | for uppercase-D "Deaf culture" people to decline these
             | kinds of treatments for their kids because they want their
             | kids to be deaf too. That's absolutely child abuse.
        
           | houseplant wrote:
           | > Deliberately restricting a human being's sense experience
           | throughout their lives should be considered child abuse
           | 
           | I've got relatives that teach deaf children. most deaf kids
           | who grow up in societies with mostly hearing kids, want to be
           | normal more than anything. At the same time, for many who are
           | eligible for cochlear implants, getting that early before
           | speech development begins in earnest is very important,
           | ideally when they're just a toddler or younger. Early enough
           | and you'll barely notice any difference in their speech,
           | understanding, etc. However, their parents often decline it
           | and say they want to let their child be the one to choose
           | when they're old enough to- which is far, far outside the
           | best window for developing that area of the brain, as well as
           | speech. They might be behind in those ways for the rest of
           | their lives if they're not given therapy to help catch up.
           | 
           | I understand both sides I guess, but if consent is the issue,
           | why not give them the implant and let them choose to
           | disconnect the receiver then when they're older? Obviously,
           | because they won't want to, and rarely ever do.
        
         | lannisterstark wrote:
         | >not unlike queer people and the attempts to "fix" them.
         | 
         | To be fair, LGBTQ community isn't 'broken.' Some people are
         | inherently gay, lesbian, etc.
         | 
         | Deafness _is_ an actual defect. The weird thing that we should
         | not strive to fix human defects when they are truly defects is
         | astounding.
        
           | alexey-salmin wrote:
           | Some people are inherently deaf, what's the difference?
        
             | Veraticus wrote:
             | Being LGBTQ is not a disability or handicap; it's simply
             | different, like being red-haired or left-handed.
        
               | alexey-salmin wrote:
               | What makes you think that deafness is not the same as
               | being left-handed?
               | 
               | I mean objectively, not based on the current cultural
               | norms which will be very different 50 years from now.
        
               | Veraticus wrote:
               | "Objectively," left-handed versus right-handed changes
               | nothing about a person's capabilities in the world,
               | whereas being deaf does.
               | 
               | Cultural norms is an interesting comparison. Despite
               | there being no actual difference in capacity, cultural
               | views forced many left-handed people to be right-handed,
               | making those people miserable in the process for no good
               | reason.
        
               | alexey-salmin wrote:
               | Being deaf is surely different from not being deaf but
               | you can't objectively say it's a disadvantage. It's only
               | a disadvantage in a world built for not deaf people, like
               | being left-handed is a disadvantage in a world built for
               | right-handed people.
        
               | Veraticus wrote:
               | I can and did say that. The world is easier for non-deaf
               | people. I can't imagine even many deaf people would argue
               | against that statement.
               | 
               | The correct way to argue against this isn't to say that
               | "objectively" the world is the same for deaf and non-deaf
               | people; it's that there's a culture and language bound up
               | in deafness that don't deserve to die thanks to medical
               | advances. That is true, and makes treatments like these
               | and what they mean to the deaf community much more
               | complicated and difficult.
        
               | alexey-salmin wrote:
               | If dolphins could communicate with us they would surely
               | tell how much easier it is when you have ultrasonic
               | hearing. All humans are therefore disabled and in need of
               | fixing.
               | 
               | Or maybe not, if dolphins turn to be wiser than an
               | average human.
        
               | sweezyjeezy wrote:
               | If all humans could echo-locate except a subset who
               | couldn't, I would say that group is at a small
               | disadvantage, yes, because the set of things the main
               | group can do is 'objectively' greater. I don't know how
               | relevant it would be for us especially in the daytime,
               | but hey.
        
               | alexey-salmin wrote:
               | I agree. However, whether all kids should receive gene
               | therapy to develop the echo-location once the science
               | allows for that is a more nuanced question.
        
               | geraldwhen wrote:
               | If most buildings lacked light because the vast majority
               | just echolocated, then the those unable would be
               | disabled.
               | 
               | It is okay for there to be a normal human experience, and
               | define inability to participate as a disability.
        
               | alexey-salmin wrote:
               | > It is okay for there to be a normal human experience,
               | and define inability to participate as a disability.
               | 
               | That's what most people normally do, yes. Then many
               | people out there define an "ability to enjoy hetero sex"
               | as a "normal human experience" and therefore see gayness
               | as a disability that needs a cure.
               | 
               | I'm not arguing about the conclusion here, but about the
               | method and the basis for deriving this conclusion.
               | 
               | The initial comment in this thread declared deafness to
               | be an "actual defect" while gayness "is just people being
               | inherently gay". Such division is completely arbitrary
               | and doesn't follow from any law of nature. Only from
               | current societal views which change a lot with time.
        
               | sweezyjeezy wrote:
               | > doesn't follow from any law of nature
               | 
               | Natural selection gave us hearing.
        
               | alexey-salmin wrote:
               | Right, and it also gave us a strong desire for the
               | opposite sex. So if you draw the line based on this
               | principle then gayness and deafness will fall on the same
               | side of this line, whichever side it is.
               | 
               | Hence I write that the initial comment making the
               | distinction between gayness being obviously OK and
               | deafness being obviously not OK, look arbitrary to me.
               | This division is cultural.
        
               | sweezyjeezy wrote:
               | This feels a bit tenuous. What world do you envisage
               | where it's a completely level playing field? Do we ban
               | talking, music, sound in movies etc etc??
        
               | alexey-salmin wrote:
               | I think building a completely level playing field is a
               | dangerous utopia. Essentially it's the same idea as
               | fixing people to make them equal, just addressed from the
               | opposite end.
        
               | PierceJoy wrote:
               | It's a disadvantage in the "built" world, but it's also a
               | disadvantage in the natural world. If you drop a deaf
               | person in the middle of the forest with no one around,
               | they will not be able hear rivers for water, they will
               | not be able to hear animals approaching, etc.
               | 
               | Being deaf is objectively a disadvantage because 4 senses
               | is objectively worse than 5 senses.
        
               | alexey-salmin wrote:
               | Most of vertebrates (including human's ancestors) lost
               | the 6th sense of electric fields in process of the
               | evolution. Apparently 6 is not always better than 5 from
               | the nature's point of view. Moles went further to loose
               | sight as well.
        
               | PierceJoy wrote:
               | > Most of vertebrates (including human's ancestors) lost
               | the 6th sense of electric fields in process of the
               | evolution.
               | 
               | Human ancestors are not human. Also, the ancestor you are
               | referring to was a fish, and could only sense electric
               | fields under water. Why would the ability to sense
               | electric fields under water be an evolutionary advantage
               | for humans who don't live in water? If it's not an
               | evolutionary advantage, then there is no reason it would
               | propagate.
               | 
               | When I said 5 senses are better than 4, I was clearly
               | referring to senses that are useful in our environment.
               | Answer this question: is a deaf person, all else equal,
               | more likely or less likely to survive and procreate
               | relative to a hearing person?
               | 
               | > Moles went further to loose sight as well.
               | 
               | Moles have eyes and can see. Their vision is just not as
               | detailed as humans. Highly detailed sight isn't an
               | advantage for creatures that live in the dark. However,
               | hearing is a huge advantage for people who live in an
               | environment where sound waves exist.
               | 
               | You're clearly wrong here. Stop reaching so hard and just
               | move on.
        
               | alexey-salmin wrote:
               | No, I'm not wrong, I just don't like relying on "obvious"
               | statements like "5 senses are better than 4". We can't
               | know for sure what was "better" until the human race
               | experiment is finished (and then we won't know either
               | obviously).
               | 
               | Consider this: hereditary autoimmune diseases are usually
               | seen as a disadvantage. However they were a huge
               | advantage during the bubonic plague in Europe, increasing
               | the chances of survival by estimated 40% [1]
               | 
               | If we manage to eradicate these disadvantageous genes we
               | may not survive the next pandemic. I don't have the
               | knowledge to predict whether deafness genes or some other
               | property entangled with them will be advantageous 10000
               | years from now and neither do you. That's all. Now you
               | can enjoy listening to music all you like, it's just
               | beyond the point.
               | 
               | [1] https://www.nih.gov/news-events/nih-research-
               | matters/how-bla...
        
               | PierceJoy wrote:
               | Hereditary autoimmune diseases are not a sense, and are
               | irrelevant to this discussion about whether hearing is an
               | objective advantage or not.
               | 
               | I'll ask again, since you must have missed this question:
               | is a deaf person, all else equal, more likely or less
               | likely to survive and procreate relative to a hearing
               | person?
        
               | alexey-salmin wrote:
               | > I'll ask again, since you must have missed this
               | question: is a deaf person, all else equal, more likely
               | or less likely to survive and procreate relative to a
               | hearing person?
               | 
               | You know the answer: it can be both ways depending on
               | circumstances.
               | 
               | * In the prehistoric world I think he was less likely to
               | survive. The difference doesn't seem to be dramatic
               | though since these genes were not eradicated from the
               | population.
               | 
               | * In the modern world the difference is close to zero
               | with an unknown sign. Given that in developed countries
               | probability to procreate seem to be limited by a desire
               | to procreate, I can't rule out that e.g. deaf people for
               | some reason have 0.1% more desire to have kids, or any
               | other side-effect. So answer to your question is unknown,
               | requires a study to figure this out.
               | 
               | * In the future hearing can be an advantage or a
               | disadvantage depending on how the circumstances evolve.
               | We see that species gain and loose senses depending on
               | the environment.
               | 
               | EDIT: I would also like to clarify this part:
               | 
               | > Hereditary autoimmune diseases are not a sense, and are
               | irrelevant to this discussion about whether hearing is an
               | objective advantage or not.
               | 
               | It's a human trait which was "obviously good" in the past
               | and is "obviously bad" now. Hence I don't trust
               | statements that other human traits like deafness are
               | obviously good or bad. It's interesting to discuss but
               | it's not granted.
        
               | glenstein wrote:
               | I don't think that's quite right, evolution makes trade
               | offs and allocates scarce resources, it's not necessarily
               | because such things would not be beneficial.
        
               | alexey-salmin wrote:
               | It's not clear what "beneficial" means if you consider it
               | separately from the resources required to achieve it.
               | 
               | E.g. it's beneficial to be stronger I guess, and gorillas
               | are always strong whether they use their muscles or not.
               | However for humans nature chose a different path where
               | only the muscles you actively use are strong. This
               | approach seem to work great so far, even though it
               | results in many individual muscles of the body being
               | weak. For each and every of these muscles you can argue
               | that making it stronger would be "beneficial" but as a
               | whole it doesn't seem to work out.
        
               | RamblingCTO wrote:
               | How do you hear predators approaching? It's an objective
               | disadvantage.
        
               | herbst wrote:
               | Interestingly enough it was still common to train left
               | handed kids in right handed writing up until maybe 40
               | years ago as it was seen as some kind of defect
               | obviously.
        
               | rjh29 wrote:
               | You can't procreate, that sounds like a pretty big
               | difference in capacity?
        
               | Drakim wrote:
               | Gay people can obviously procreate, nothing about your
               | body stops functioning when you are gay.
               | 
               | What you mean is that they don't have sexual attraction
               | towards people of an opposite gender, where if they had a
               | relationship with them, that relationship would encourage
               | procreation.
               | 
               | To me that just doesn't seem like a "difference in
               | capacity".
               | 
               | I personally don't want children, and will probably seek
               | out a partner who likewise does not want children. If I
               | encounter somebody who wants lots of children, I will see
               | that we have different life goals, and I probably won't
               | be very keen on being in a relationship with them. Do you
               | consider me "damaged" or of "diminished capacity" because
               | of it?
        
               | ben_w wrote:
               | > "Objectively," left-handed versus right-handed changes
               | nothing about a person's capabilities in the world,
               | 
               | As a leftie, this is only true in the current world -- I
               | live in a culture with a left-to-right writing system,
               | and yet technology means I don't ever need to use a
               | fountain or quill pen.
               | 
               | I did have one teacher who insisted on "no biro" when I
               | was a kid, but they were also my first introduction to
               | "not everyone is actually nice".
        
               | stahtops wrote:
               | Let me help explain, some left handed people can hear and
               | are not deaf. Objectively that is how deafness is not the
               | same as being left-handed.
        
               | alexey-salmin wrote:
               | Well thank you, but the question is why one needs
               | "fixing" and the other doesn't. Parent comment made an
               | argument that being gay is like being left-handed so it
               | doesn't need "fixing". That's fine with me, but I don't
               | see how the same logic doesn't apply to deafness.
               | 
               | Many deaf people enjoy their life as it is and don't
               | welcome your attempts to "fix" them.
        
               | stahtops wrote:
               | I didn't attempt to "fix" anyone. Everyone has to do that
               | themselves.
               | 
               | What about the deaf people that don't enjoy their life as
               | it is and do welcome the opportunity to hear?
        
               | alexey-salmin wrote:
               | > What about the deaf people that don't enjoy their life
               | as it is and do welcome the opportunity to hear?
               | 
               | I would be happy if they had such an opportunity.
               | 
               | The question, once again, was different: should the
               | hereditary deafness be eradicated in childhood by gene
               | therapy once advances of medicine allow that?
        
               | glenstein wrote:
               | I think you changed the question and escalated the
               | severity of the proposal so it became easier to knock
               | down as a straw man.
               | 
               | Originally this was about the desirability and capability
               | of enabling deaf people to hear writ large. But now you
               | are framing it as full scale eradication carried out in a
               | way that bypasses consent.
        
               | alexey-salmin wrote:
               | I'm not, please read the whole discussion.
               | 
               | The root of this thread referenced the cochlear implant
               | which produces best results when implanted at a very
               | young age (staring from 9 months) obviously without
               | consent of the patient.
               | 
               | Other author replied that:
               | 
               | > The weird thing that we should not strive to fix human
               | defects when they are truly defects is astounding.
               | 
               | All my comments are essentially stating the disagreement
               | (or rather lack of agreement) with this point of view.
               | 
               | It declares being deaf as a "true defect" that we should
               | "strive to fix". In the context of the gene therapy now
               | available, I understand this means that a deaf-born
               | children should be "fixed" to remove this "defect". This
               | vision is not crazy, but it doesn't strike me as
               | universally true either.
        
               | glenstein wrote:
               | I read it multiple times and the unresponsiveness of this
               | comment serves to further confirm my assessment, that you
               | were unique in introducing the notion of full-scale
               | eradication without qualification into this conversation.
               | The participant you quoted noted a preferability, but you
               | engaged in an original creative act in equating this to
               | eradication.
        
               | magicalhippo wrote:
               | > The question, once again, was different: should the
               | hereditary deafness be eradicated in childhood by gene
               | therapy once advances of medicine allow that?
               | 
               | Sure, because it's reversible. If they don't like hearing
               | they can always become deaf again.
        
               | geraldwhen wrote:
               | If you feel compelled to remove parts of your body and
               | take opposite sex hormones for life, that's clearly a
               | disability.
               | 
               | If genes could fix your brain to not feel compelled to
               | swap sexes, that would be a huge win in the quality of
               | life for most individuals.
               | 
               | You can pretend that pretty much anything "isn't a
               | disability, it's just different." But that isn't true.
        
               | seattle_spring wrote:
               | You're focusing pretty hard on a single letter of that
               | acronym that makes up a small percentage of the overall
               | community.
        
         | ugh123 wrote:
         | That would be a terrible reason to give up, or slow down, on
         | research in any area of health science.
        
       | 911e wrote:
       | How likely to cure tinnitus ?
        
         | tux3 wrote:
         | Zero chance, unfortunately. The cause of tinnitus is not with
         | the OTOF gene.
         | 
         | The best case scenario is that this wouldn't do anything.
        
           | ck_one wrote:
           | There is also active development for tinnitus/noise induced
           | hearing loss. Dr Chen from Harvard is working on it. He was
           | also involved in the research linked in the article. They are
           | getting closer to a cure. Here is an interview with him:
           | https://www.youtube.com/watch?v=lJr86MUYJ8M
        
       | carterschonwald wrote:
       | This is the sort of stuff I conceptualize as a true
       | tech(nical/nology)startup. I realize that's in some ways at odds
       | with the meaning in practice. But this is amazing technology.
        
         | GOD_Over_Djinn wrote:
         | Discoveries like this should be shared with the world - not
         | gatekept by rent-seeking venture capitalists. Wouldn't you
         | rather be Jonas Salk than Scrooge McDuck?
         | 
         | https://en.wikipedia.org/wiki/Jonas_Salk
         | 
         | > Salk was immediately hailed as a "miracle worker" when the
         | vaccine's success was first made public in April 1955, and
         | chose to not patent the vaccine or seek any profit from it in
         | order to maximize its global distribution
        
           | chris_t wrote:
           | Rent-seeking has a technical definition; earning money from
           | things you invented does not meet it. While Salk is obviously
           | an inspiring example, a wise society should happily
           | incentivize the development of useful technology.
        
             | carterschonwald wrote:
             | Well said
        
             | sooheon wrote:
             | Intellectual monopolies disincentivize development and
             | incentivize rent seeking. Here, rent seeking is legal
             | action and lobbying intended to protect and extend
             | monopolies, instead of advancing science.
             | 
             | Other than economic logic, we have natural experiments to
             | back this up: i.e. the steam engine patent granted to Watt
             | and Boulton in 1772 and its chilling effect on engine duty
             | improvement (and the explosion of progress after patent
             | expiry), and the modern pharmaceutical industry developing
             | most strongly precisely where chemical patents were not
             | granted, in 19th C. continental Europe, and slowing down
             | wherever patent protections were eventually introduced, at
             | the request of rent seeking lobbyists.
             | 
             | https://fee.org/articles/do-patents-encourage-or-hinder-
             | inno...
             | 
             | http://www.dklevine.com/papers/anew09.pdf
        
               | chris_t wrote:
               | I agree with all of this. The example of the patent on
               | steam engines is fascinating, thank you! It's certainly
               | true government granted monopolies are far from the ideal
               | incentive structure, with many downsides. (And lobbying
               | for EG patent extensions likely IS rent-seeking). Advance
               | market commitments and other systems likely have better
               | characteristics.
               | 
               | I was rather objecting to the misuse of a technical term,
               | and the idea that we should expect all innovation to
               | happen without reasonable incentives.
        
       | arjie wrote:
       | Through a mutual acquaintance, I met someone who did a little
       | work for on the OTOF gene therapy and it really is remarkable
       | stuff.
       | 
       | My wife and I had our embryos screened by Orchid Health since we
       | have a related genetic condition: a pathogenic mutation in GJB-2.
       | Amazingly, Decibel Therapeutics has has one of these in R&D and
       | the OTOF success gives us hope. We have a few embryos that are
       | affected and the more that can grow up healthy the more chances
       | we'll have at children.
       | 
       | The technology itself is unbelievably futuristic: involving using
       | a virus to deliver a repaired gene into existing cells.
       | 
       | This form of non-syndromic hearing loss may well be repaired two
       | generations from now. An interesting thing is if you follow along
       | with the literature, some Chinese labs report success with these
       | gene therapies in kids on the cusp of teen age. That's
       | remarkable. It will give a lot of people today the chance to have
       | healthy kids in the future even if they were unfortunate enough
       | to carry the genes today.
       | 
       | Personally, the fact that I can practically hold a whole genome
       | sequence of our embryos on my computer means the future is here.
       | Last time, I posted a few links for everyone interested in the
       | subject https://news.ycombinator.com/item?id=40312242
        
         | SJC_Hacker wrote:
         | Screening can also "repair" inherited deafness through
         | selection - you simply don't produce offspring to those that
         | have the defect.
        
       | nehagup wrote:
       | When I try and imagine what that must be like from the inside--I
       | can't think of anything more exciting or scary than to gain a new
       | sense.
        
         | herbst wrote:
         | There are magnet Implantats you can put into your finger which
         | lets you feel magnetic fields.
        
           | meindnoch wrote:
           | That's not a new sense, just using the existing tactile
           | system with a magnet.
        
             | iandanforth wrote:
             | Senses are defined by what they sense, not how they are
             | sensed. A person can have their sight partially restored
             | through implants that bypass rod/cone activation.
             | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6934168/
        
             | knodi123 wrote:
             | you'd be surprised.
             | 
             | I've heard about people experimenting by wearing a belt
             | with dozens of haptic motors and a compass, so that it
             | always indicates the direction of north. After a while
             | wearing it, they stop consciously feeling the sensation of
             | the vibration, and just feel "northness". And then when
             | they take off the belt, they feel weirdly disoriented for a
             | while, even in familiar places.
        
         | LadyCailin wrote:
         | There is a belt you can wear that has vibrators around it, and
         | it always vibrates the north facing side. I read that people
         | who wore that for a few weeks started gaining the ability to
         | create much more accurate maps in their head, which makes
         | sense, this is how pigeons do it too, they basically can just
         | always sense where north is.
        
       | nehagup wrote:
       | If I'm interpreting the article correctly, it seems like the
       | amount of hearing recovered is pretty good. I was wondering how
       | much hearing was actually restored.
        
       | 29athrowaway wrote:
       | The genetic corrections are not inherited, right?
        
         | zac256 wrote:
         | It's considered taboo/forbidden for genetic corrections to
         | target the germline. I think that's an unfortunate
         | misapplication of ethics, presumably due to fears about
         | eugenics.
        
           | BurningFrog wrote:
           | It's taboo in Western medicine.
           | 
           | I doubt the whole world will respect that.
        
           | djur wrote:
           | My understanding is that it's more about mitigating risk than
           | avoiding an association with eugenics. If the gene therapy
           | has unforeseen consequences the harm is multiplied with every
           | generation and is harder to address once found.
        
       | myrloc wrote:
       | How can I get involved in this work as a software engineer?
        
         | rjh29 wrote:
         | If you can't find out yourself then you're probably not
         | motivated enough.
        
           | myrloc wrote:
           | Does asking the HN community not fall into the category of
           | "finding out"?
        
           | bedobi wrote:
           | What an uncharitable thing to say
        
         | MrDresden wrote:
         | Having worked at a large world renowned genetics laboratory as
         | an SWE I believe I can say that it is unlikely you will have
         | the option of having any direct input into this kind of work,
         | in that role.
         | 
         | That being said, there is a lot of things an SWE can do in a
         | support role.
         | 
         | Some of the things I did were data processing pipelines (both
         | automatic and human centric UI for phenotype gathering) and
         | interconnected networks of scientific instruments, as well as
         | some deployments of R and Python code.
         | 
         | Sure, your name wont show up along with hundreds of others in
         | the Nature publications, but you will know that you contributed
         | to the overall success. And it does feel good.
        
       | jdthedisciple wrote:
       | > Two of the children even gained an ability to appreciate music.
       | 
       | Interesting: Does this mean that the other 3 children are still
       | kind of indifferent to Music and presumably other kinds of
       | sounds?
        
         | meindnoch wrote:
         | There's a certain age beyond which you cannot learn proper
         | speech. There's a certain age beyond which you cannot learn
         | absolute pitch.
         | 
         | It wouldn't be surprising if there was a certain age beyond
         | which you cannot learn to understand music.
        
           | CaptainOfCoit wrote:
           | > There's a certain age beyond which you cannot learn
           | absolute pitch
           | 
           | I can understand things like that get harder and harder as
           | you become adult. That it's absolutely not possible beyond a
           | certain age I think is just a commonly held belief that
           | doesn't hold up to scrutiny.
           | 
           | What is this age limit where you've never seen anyone be able
           | to gain that skill? I'm sure there are counter-examples.
        
             | rylittle wrote:
             | Interestingly enough, I once read that no one over the age
             | of 60 has ever _retained_ perfect pitch
        
       | deepGem wrote:
       | "The children involved in the study were born with DFNB9, which
       | accounts for between 2 and 8% of hereditary deafness. It is
       | caused by mutations in the OTOF gene which prevent the production
       | of functioning otoferlin protein, which can significantly reduce
       | sound transmission from the inner ear hair cells to hearing
       | nerves."
       | 
       | Just shows how much of our body functions we take for granted.
       | Every function has a critical path that is so vulnerable. This is
       | despite the human body having all kinds of redundancies. Our body
       | is so resilient, yet at times, some of the mechanisms are so
       | fragile.
        
         | herodoturtle wrote:
         | Poignant observation (that we take so much of our bodily
         | function for granted), thank you for adding this to the
         | commentary on this page.
         | 
         | And that resilience-with-fragility you speak of instantly
         | reminded me of our giant cloud clusters, with their tiny points
         | of failure that test the limits of redundancy.
        
       | belter wrote:
       | "Aissam, the boy who escaped from a world of silence thanks to
       | gene therapy": https://english.elpais.com/science-
       | tech/2024-06-05/aissam-th...
        
       | BurningFrog wrote:
       | Language nit:
       | 
       | You can't "restore" hearing to those who never had it.
        
         | ADeerAppeared wrote:
         | That's not just a nit.
         | 
         | The idea that these are people who are "broken" and need to be
         | "fixed" is something a lot of Deaf people are very opposed to.
        
           | CaptainOfCoit wrote:
           | > The idea that these are people who are "broken" and need to
           | be "fixed" is something a lot of Deaf people are very opposed
           | to.
           | 
           | Are they maybe "opposed" to been seen like that because there
           | was no hope for any solutions besides just being OK with it?
           | 
           | In the future when it's more accessible and cheaper,
           | shouldn't being born without hearing be considered a "fault"
           | of sorts, as you can now introduce this sense to them, like
           | most others being born with hearing at birth?
        
       | very_good_man wrote:
       | All of us have to get real and demand immediate speed-up of LLMs,
       | Gene Therapy, etc. Absolutely nothing else matters.
        
         | arnaudsm wrote:
         | Millions of people already die of curable diseases. Technology
         | isn't enough, many problems are political
        
         | bn-l wrote:
         | Fully agree. Both have the potential to solve whole groups of
         | problems. Gene therapy especially should be species goal as
         | something to get us to the next stage of evolution.
        
         | bluerooibos wrote:
         | Politics could do with an injection of LLM action. Climate
         | change is arguably the most urgent issue.
        
       | throwaway48476 wrote:
       | Genetic issues are more prevalent in communities that practice
       | inbreeding. Eliminating inbreeding seems to be an easier fix than
       | genetic therapy.
        
       | nobodyandproud wrote:
       | Tangent, but this reminds me of
       | http://news.bbc.co.uk/2/hi/health/1916462.stm
        
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