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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 relat
by DubiousPusher 6y ago
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 https://www.nature.com/articles/s41591-020-0763-1
There are currently active stage 2 trials for multiple forms of achromatopsia.
- mhneu 6y agoYes, 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/d43412c1709861aea218e4cba2240921668e.pdf?_ga=2.152739449.1717129560.1603239977-716216739.1603239977 https://pdfs.semanticscholar.org/cba7/d43412c1709861aea218e4...
- Areading314 6y agoThey are also doing trials for a CRISPR-based gene therapy solution: https://ir.editasmedicine.com/news-releases/news-release-details/allergan-and-editas-medicine-announce-dosing-first-patient https://ir.editasmedicine.com/news-releases/news-release-det...
- jiofih 6y ago> As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology It’s like neither of you read the article.
- Areading314 6y agoIn humans
- thaumasiotes 6y ago> 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.
- TylerE 6y agoOcular 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.
- DubiousPusher 6y agoI 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.
- refurb 6y agoMacular degeneration is very common with 11M people in the US affected. It’s treated with intraoccular injections of VEGF inhibitors.
- DoingIsLearning 6y agoNope, 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.
- tarboreus 6y agoSome new therapies just inject a virus into the vitreous.