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Paper Details

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.
Nat Med
399
2019
CEP290, CEP290 gene, CEP290 gene1, CRISPR, Cas9, EDIT, EDIT-, EDIT-101, IVS26 mutation, LCA10, Leber congenital amaurosis type 10, NHP CEP290 gene, Staphylococcus aureus, Staphylococcus aureus Cas9 guide RNAs, aberrant splice donor, human, human CEP290 target sequence, human cells, humanized, inherited retinal disorders, mice, retinal dystrophy, somatic primate cells, vision loss

Datasets

Release 1.2