Article

Gene augmentation therapy treats mature mice with complete congenital stationary night blindness (cCSNB), improving retinal function and visual acuity

2025-07-31

Abstract excerpt

<h4>ABSTRACT</h4> Recombinant adeno-associated virus (rAAV) mediated gene therapy is an effective approach for targeting therapeutic genes to retinal photoreceptors. Complete congenital stationary night blindness (cCSNB) is a genetically heterogeneous inherited retinal disease caused by mutations in one of several genes, which are part of a large and interdependent depolarizing bipolar cell (DBC) signalplex requi...

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Literature Corpus work: 6eed52b8-cf85-5717-845d-22052a8c86df
DOI: 10.1101/2025.07.25.666812

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