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Deaf girl is cured in world first gene therapy trial (www.independent.co.uk)

submitted 6 months ago by [email protected] to c/[email protected]

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Opal Sandy, aged 18 months, was born completely deaf due to condition auditory neuropathy

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[–] [email protected] 0 points 6 months ago* (last edited 6 months ago) (1 children)

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

[–] [email protected] 0 points 6 months ago (2 children)

So, the cure is hereditary?

[–] [email protected] 0 points 6 months ago

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.

[–] [email protected] 0 points 6 months ago

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.