Deaf girl is cured in world first gene therapy trial
Opal Sandy, aged 18 months, was born completely deaf due to condition auditory neuropathy
Opal Sandy, aged 18 months, was born completely deaf due to condition auditory neuropathy
Great achievement. Not much on the tech though. According to the ucam press release, they used an AAV1 viral delivery system. Given that the therapy is permanent, I would expect integration of the therapeutic gene into the patient's genome (in the targeted tissue). Anyone have more on this?
https://www.cam.ac.uk/research/news/baby-born-deaf-can-hear-after-breakthrough-gene-therapy
Edit: I think this patent may be relevant
https://www.patentsencyclopedia.com/app/20130095071
So, the cure is hereditary?
No
The gene therapy is applied locally. They operate on the ear to enable access to the relevant tissue and then apply the therapy. So it should only affect cells in the ear.
Additionally, it is unclear to me whether the therapeutic gene is integrated into the genome. It might not be.
For transmission to offspring, you would need to edit germ line cells (sex cells) and you would need to integrate the gene into the genome.
I don't know anything about the treatment. But to be hereditary the genome changes would have to affect gametes. If genes are only changed in other targeted tissue then the changes wouldn't be inherited.
