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Re:Study reveals restoration of retinal and visual function ... by DubiousPusher on Tuesday October 20, 2020 @07:56PM (#24843548)
Gene therapy for retinal disorders is kind of low hanging fruit in the gene therapy world because the affected cells are few in number, well contained and relatively accessible (despite needing to stick needle into the patients eye).

There is already an FDA approved treatment for one heritable form of retinitis pigmentosa.

https://www.nature.com/articles/s41591-020-0763-1

There are currently active stage 2 trials for multiple forms of achromatopsia.

Re:Study reveals restoration of retinal and visual function ... by mhneu on Tuesday October 20, 2020 @08:27PM (#24843733)
Yes, it is the low hanging fruit.

The risk is also mitigated because if the gene manipulation causes cancer, it should be relatively restricted to the eye, which can be removed in the worst case.

For a long time, gene therapy was shied away from because of an early case of oncogenesis with a lentiviral vector: https://pdfs.semanticscholar.org/cba7/d43412c1709861aea218e4...

Re:Study reveals restoration of retinal and visual function ... by Areading314 on Tuesday October 20, 2020 @09:59PM (#24844163)
They are also doing trials for a CRISPR-based gene therapy solution: https://ir.editasmedicine.com/news-releases/news-release-det...
Re:Study reveals restoration of retinal and visual function ... by jiofih on Wednesday October 21, 2020 @02:43AM (#24845323)
> As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology

It’s like neither of you read the article.

Re:Study reveals restoration of retinal and visual function ... by Areading314 on Wednesday October 21, 2020 @12:56PM (#24849690)
In humans
Re:Study reveals restoration of retinal and visual function ... by thaumasiotes on Tuesday October 20, 2020 @10:45PM (#24844308)
> relatively accessible (despite needing to stick needle into the patients eye)

At least this requirement is much less nasty in a treatment for blindness than it would be in anything else.

Re:Study reveals restoration of retinal and visual function ... by TylerE on Wednesday October 21, 2020 @12:51AM (#24844829)
Ocular injections aren’t that uncommon.

Done before every lasix procedure, for instance. I don’t think it’s supposed to be that painful (there aren’t many nerves in the eye), just freaky. I believe oral sedatives are common.

Re:Study reveals restoration of retinal and visual function ... by DoingIsLearning on Wednesday October 21, 2020 @02:29AM (#24845262)
Nope, this is definitely a retinal surgery. Sub-retinal injections are actually done through the inside of the eye.

Your eyeball is filled with vitreous humour which although technically a fluid is really like a thick layer of gelatine, surgical instruments can't navigate that.

Before a needle is able to reach that retinal area, the surgeon must perform what is known as a Vitrectomy which is basically a chop and suction of that gelatine in order to have a good visual and be able to accurately manipulate surgical instruments.

Source: worked in embedded software for ophthalmic surgery equipment.

Re:Study reveals restoration of retinal and visual function ... by tarboreus on Wednesday October 21, 2020 @12:50PM (#24849606)
Some new therapies just inject a virus into the vitreous.
Re:Study reveals restoration of retinal and visual function ... by DubiousPusher on Wednesday October 21, 2020 @01:05AM (#24844899)
I don't think this is comparable. The needle must reach the retina. I am no expert but I recall the phase one trials were done to merely test if the vector was safe.
Re:Study reveals restoration of retinal and visual function ... by refurb on Wednesday October 21, 2020 @02:44AM (#24845327)
Macular degeneration is very common with 11M people in the US affected. It’s treated with intraoccular injections of VEGF inhibitors.