Article

A gene-augmentation framework for selected early-stage autosomal recessive retinitis pigmentosa genotypes

2026-07-26

Abstract excerpt

Many recessive inherited retinal dystrophies are, in principle, amenable to gene augmentation, yet only one such therapy has received regulatory approval. We sought to identify potentially tractable autosomal recessive retinitis pigmentosa (RP) genotypes that might be addressed using publicly described adeno-associated virus (AAV) components. More than 100 RP-associated genes were prioritized according to cellular...

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Literature Corpus work: ac21cd51-591e-5c1f-8157-da3b651f59e3
DOI: 10.64898/2026.07.25.740701

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