Article

Nonviral PDMAEMA Vectors Efficiently Express PDE6β in a Mouse Model of Retinitis Pigmentosa

2023-05-09

Abstract excerpt

Retinitis Pigmentosa (RP) is one of main causes of inherited blindness, with about 6% of cases caused by a single mutation in the PDE6β gene, making it an ideal candidate for a gene therapy intervention. Gene therapy has been shown to restore normal retinal and visual function in other monogenic diseases, such as Lebers’ Congenital Amaurosis and choroideremia, and RP could benefit from a similar therapeutic approa...

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Literature Corpus work: be92f7b8-2219-5703-9410-14003a06b808
DOI: 10.1101/2023.05.04.538567

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