Article

High-efficiency base editing for Stargardt disease in mice, non-human primates, and human retina tissue

2023-04-17

Abstract excerpt

<h4>ABSTRACT</h4> Stargardt disease is a currently untreatable, inherited neurodegenerative disease that leads to macular degeneration and blindness due to loss-of-function mutations in the ABCA4 gene. We have designed a dual adeno-associated viral vector split-intein adenine base-editing strategy to correct the most common mutation in ABCA4 (c.5882G>A, p.G1961E). We optimized ABCA4 base editing in human model...

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Literature Corpus work: 2033392f-1f86-5923-9b46-f4b6be7d9ea5
DOI: 10.1101/2023.04.17.535579

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