Article
Allele-specific gene editing to rescue dominant CRX-associated LCA7 phenotypes in a retinal organoid model.
Stem cell reports - 9 Nov 2021
Chirco Kathleen R, Chew Shereen, Moore Anthony T, Duncan Jacque L, Lamba Deepak A
Abstract excerpt
Cases of Leber congenital amaurosis caused by mutations in CRX (LCA7) exhibit an early form of the disease and show signs of significant photoreceptor dysfunction and eventual loss. To establish a translational in vitro model system to study gene-editing-based therapies, we generated LCA7 retinal organoids harboring a dominant disease-causing mutation in CRX. Our LCA7 retinal organoids develop signs of immature...
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