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Small molecule treatment alleviates photoreceptor cilia defects in LCA5-deficient human retinal organoids

2025-02-17

Abstract excerpt

<title>Abstract</title> <p>Bialleleic pathogenic variants in <italic>LCA5</italic> cause one of the most severe forms of Leber congenital amaurosis, an early-onset retinal disease that results in severe visual impairment. Here, we report the use of gene editing to generate isogenic <italic>LCA5</italic> knock-out (LCA5 KO) induced pluripotent stem cells (iPSC) and their differentiation to retinal organoids. The m...

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Literature Corpus work
9dea5cc3-298b-5ee4-ac16-371110f1b1e7
DOI
10.21203/rs.3.rs-5219388/v1
Open publication

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Small molecule treatment alleviates photoreceptor cilia defects in LCA5-deficient human retinal organoidsDOI 10.21203/rs.3.rs-5219388/v1
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