Current perspectives in Leber congenital amaurosis type 8 mouse modeling
Sign Up to like & getrecommendations! Published in 2022 at "Developmental Dynamics"
DOI: 10.1002/dvdy.462
Abstract: Mutations in the CRB1 (Crumbs homolog 1) cause rare retinal diseases like retinitis pigmentosa type 12 (RP12) and Leber congenital amaurosis type 8 (LCA8). RP12 results in progressively worsening peripheral vision, whereas LCA8 causes severe… read more here.
Keywords: leber congenital; amaurosis type; type; congenital amaurosis ... See more keywords
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
Sign Up to like & getrecommendations! Published in 2019 at "Nature Medicine"
DOI: 10.1038/s41591-018-0327-9
Abstract: Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the CEP290 gene1,2. We developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor created by the IVS26 mutation… read more here.
Keywords: gene editing; amaurosis type; congenital amaurosis; gene ... See more keywords