I expect you will have seen this AAV gene therapy for autosomal recessive deafness 9: a single-arm trial | Nature Medicine
It could be very good news for children and young adults with hearing loss. I wonder if gene therapy could help with age related hearing loss. I expected to see a thread here about this research so that’s why I started this one.
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After Frequency Therapeutics, it’s something we stear away from talking about. Anyway, not gene therapy maybe, but you might want to look up Rinri Therapeutics. They’ve recently been approved by the UK equivalent of the FDA to conduct clinical trials and they expect to present data 12 months after they commence. Presbycusis (age-related hearing loss) is the main unmet need they’re trying to find treatments for.
What I like about them is that they still have links to the UK universities where the research originated and they aren’t as manically self-promoting as Frequency Therapeutics were.
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Any reason “we” are not interested in AAV-mediated gene delivery?
I was talking about cures or treatments for hearing loss in general, and it was just an observation. There was a lot more talk about potential treatments for hearing loss prior to Frequency Therapeutics. That debacle seemed to sour people on the possibility of medical intervention and it’s been rarely mentioned since. I’m not trying to control what you’re interested in.
OP is interested in presbycusis. I haven’t seen anything to suggest that gene therapy has a role to play but I’m happy to be educated on that.
Ah, thx for explaining. Yes, anything involving “genes” or “viruses” may lead to panic among some…
The inner ear is actually one of the few organs that is quite accessible to gene therapy, and the safety of AAV is quite well-established, so I don’t hope the handling of this topic here will scare off investors in this area.
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Thanks d_Wooluf for this. I did look up Rinri and their surgical technique for cell therapy looks very promising. Hope for us oldies but gene therapy may also turn out to be effective treatment for the young uns. Great to see the interest in developing treatments for hearing loss.
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Just commenting. This won’t be for any kind of general hearing loss. It will be for hearing losses associated with a specific gene defect.
Maybe so but I guess general hearing loss can be genetic. It certainly seems that way in my family with four generations affected to my knowledge from my paternal grandmother’s mother down to me.
Yes, “general” hearing loss could be genetic but there are likely lots of different genes and possibly combinations of genes involved. That all needs to be sorted out before therapy can be figured out. When we read about it seems so enticingly simple. It isn’t.
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Moreover, if I remember well, the genetic payload of AAV is only some 5,000 bp, so you cannot deliver a whole bunch of genes. But let’s hope…
I hadn’t seen this but it is great for children/young adults with hearing loss. I really hope that gene therapy would be able to help with age related hearing loss. I hope that there will be threads about this in due course.
This new trial is a real breakthrough, demonstrating that you can restore the functionality of a defective gene in the inner ear, opening the way to a host of new treatments. One important caveat is that the gene must also still be relevant post-development of the cochlea (i.e., in children and even adults), and that the extent of gene replacement is sufficient. Those questions can be addressed in mouse models (“knock-out” mice that carry an artificial gene deletion).
The problem with age-related hearing loss without a clear genetic cause (a huge market!) is, first, that there seem to be no good candidate genes whose overexpression might do the job. Second, no good mouse models (these animals live only for two years or so).
Such a therapy might nevertheless come for acute noise-induced hearing damage (good mouse models there), which bears some similarity to age-related hearing loss (both affecting sensorineural cells).
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