My wife and I are selecting which embryos to implant because we have a related condition (this one is a mutation in OTOF, we have one in GJB-2). Being able to implant the embryos affected with this condition (carrying two copies of the gene - mine and my wife) would increase the number of viable embryos we have by 50%.
It won't be in time for us to actually implement unless we're down to our last few embryos through failure. We were able to detect ours because we used Orchid Health to scan the embryo genome for monogenic conditions. But it's exciting to think that novel gene therapies might be accessible if we somehow fail to implant with the other embryos we have.
All this stuff is very futuristic and it's definitely rescued us since my wife and I started dating and got married within the last year, by which time we were quite old.
Very cool stuff from Regeneron.
EDIT: I actually looked this up and got a reading list which I went through over lunch. There's quite a lot of work on this front.
Regeneron/Decibel Therapeutics have DB-OTO and are developing a GJB-2 (the one we have) gene therapy too https://www.decibeltx.com/pipeline/
There's a Chinese group that claims they had success in older patients (search for AAV1-hOTOF)
The April issue of Molecular Therapy has a few of these. For a quick read look at the Oral Abstracts from the Presidential Symposium. Molecular Therapy Vol 32 No 4S1, April 2024
It won't be in time for us to actually implement unless we're down to our last few embryos through failure. We were able to detect ours because we used Orchid Health to scan the embryo genome for monogenic conditions. But it's exciting to think that novel gene therapies might be accessible if we somehow fail to implant with the other embryos we have.
All this stuff is very futuristic and it's definitely rescued us since my wife and I started dating and got married within the last year, by which time we were quite old.
Very cool stuff from Regeneron.
EDIT: I actually looked this up and got a reading list which I went through over lunch. There's quite a lot of work on this front.
Regeneron/Decibel Therapeutics have DB-OTO and are developing a GJB-2 (the one we have) gene therapy too https://www.decibeltx.com/pipeline/
Akouos has AK-OTO https://akouos.com/our-focus/
There's a Chinese group that claims they had success in older patients (search for AAV1-hOTOF)
The April issue of Molecular Therapy has a few of these. For a quick read look at the Oral Abstracts from the Presidential Symposium. Molecular Therapy Vol 32 No 4S1, April 2024