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Article

Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4

13 Feb 2024

Abstract excerpt

Biallelic variations in the aryl hydrocarbon receptor interacting protein-like 1 ( AIPL1 ) gene cause Leber congenital amaurosis subtype 4 (LCA4), an autosomal recessive early-onset severe retinal dystrophy that leads to the rapid degeneration of retinal photoreceptors and the severe impairment of sight within the first few years of life. Currently, there is no treatment or cure for AIPL1 -associated LCA4. In...

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PMID: 38439910
DOI: 10.1016/j.omtn.2024.102148

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