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Gene therapy to treat RPE65-mediated RP is administered via ________ injection, while gene therapy to treat...

Gene therapy to treat RPE65-mediated RP is administered via ________ injection, while gene therapy to treat LHON is administered as an intravitreal injection.

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Answer #1

Leber congenital amaurosis (LCA) is caused by mutations in the RPE65 gene.
Gene therapy for LCA uses a subretinal injection of Adeno-associated viral vectors to deliver the functional copy of the RPE65 cDNA to the retinal pigment epithelial cells of the patient.


Gene therapy to treat RPE65-mediated RP is administered via SUB-RETINAL injection, while gene therapy to treat LHON is administered as an intravitreal injection.

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