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  arjie
  I’ve been following this for a personal interest. Decibel Therapeutics was working on this for quite a while with a lot of success before they were acquired by Regeneron and it was very promising but the next ones in the pipeline were personally relevant. They’re a fix for the GJB2 gene mutation that causes hearing loss. Unlike the OTOF error, this one is progressive so you have to get to it fast.My wife and I carry mutations to the gene so we’ve done preimplantation genetic testing to select the embryos that haven’t been affected and our daughter can hear just fine! We have enough unaffected embryos that we can have another child but if we can have a third we’d probably want a boy[0] and both of our male embryos are coincidentally affected. If somehow we’ve managed to delay long enough for the corresponding Decibel TX AAV.103 gene therapy to come to market, then this will be an incredible triumph of modern science and technology over nature.Here’s hoping!If you’re curious about this process, I’ve written about it here: https://wiki.roshangeorge.dev/w/IVFAnd here’s the treatment pipeline image I nicked off decibel TX’s website before they were acquired a year and a half ago https://wiki.roshangeorge.dev/w/File:Screenshot_Decibel_Tx_P...I’m super thrilled everything has gone through so fast.0: it would just be nice to have children of both genders; a weak preference - if I have 3 daughters I would be thrilled anyway
• Uncorrelated
  I'm partially deaf and I've worn hearing aids since around five years old. I used to think it didn't affect me but as I get older I increasingly suspect it's negatively impacted my life more than I thought. Just the additional difficulty understanding in noisy environments affected my social life and development, and who knows what kind of knock-on effect that's had.Anyway, any progress in treating similar conditions is great news.
• m_w_
  Although this affects a small number of people / kids per year - this is a great development. There's a related article^ which had a very moving account from a mom whose son has received this treatment.^: https://www.npr.org/2026/04/23/nx-s1-5795526/deafness-gene-t...
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  NoSalt
  This is really cool and awesome for people with this condition!Unfortunately, I have seen treatments arrive for almost every type of deafness except the one affecting me. I contracted the mumps at a very young age, approximately 5 months old, and I now have nerve deafness in one ear making me completely deaf in that ear. The ear drum still operates, as I feel pain in that ear when around extremely loud noises. I absolutely do not want a cochlear implant, so it seems I may be stuck like this for the rest of my life. :-(
• ElijahLynn
  There is a short video on this by Dr. Ben Miles that explains this really succinctly and with visuals.https://youtube.com/shorts/O8HZw4AbttQ?si=QSCjyDGEIr3Ll-5hMy short interpretation is that you basically inject a virus to mutate cells permanently and it regrows missing hair that takes sound waves and turns them into electrical impulses in the brain.Absolutely amazing!
• Aurornis
  This is a very cool example of a targeted gene therapy for a very specific type of hearing loss. As the article says it only applies to a subtype of genetic hearing loss which makes up 2-8% of genetic cases, but it’s nice to see such niche therapies being developed and approved despite the smaller number of people who could benefit. It underwent an accelerated review through a new program met to fast track treatments for rare conditions like this which would normally be difficult to get approved due to the small scale.
• locao
  Coincidentally, I'm reading this at the doctor's office reception, waiting to check my (potentially genetic) hearing loss. Hope it's something simpler, but good to know there are advances in this area.
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  neonstatic
  "These kind of genetic therapies seem to reinforce this idea of deafness being a problem in need of eradication, and that the only solution for disabled people to fully assimilate into society is through a medical intervention," says Jaipreet Virdi This just seems so incredibly stupid to me.
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  newyankee
  Very positive news. I have something similar but in a much more difficult gene with different manifestation, but at least this gives me hope that something might come up in 1-2 decades.
• GalaxyNova
  Hopefully they will soon find one for noise induced hearing loss as well.
• foobar703
  I read “Hair Loss” and got excited.
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  nsxwolf
  How is it priced? Did they use the sickle cell cure pricing model? $5 less than a cochlear implant?
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  Noaidi
  This is good news, but I still have concerns.Otoferlin [1] uses calcium as a cofactor. These mutations happen for a reason. The enzyme is not only located in the ear, but also in the brain and bone marrow [2].Will there be repercussions if the virus leaves the local area when the therapy is injected?These OTOF mutation have their highest expression in the Turkish population. Many people with other variations of this gene only experience deafness when they have a fever[3]. So in my opinion, I would like to see ten year outcomes before celebrating.[1] https://www.uniprot.org/uniprotkb/Q9HC10/entry[2] https://www.proteinatlas.org/ENSG00000115155-OTOF/tissue[3] https://www.frontiersin.org/journals/cell-and-developmental-...
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  martinbfine
  That's great! Kennedy's team is changing the world and how we see treatments. No more lifetime suppression of symptoms, more cures!