The steady progress in genome editing, especially genome editing based on the use of
clustered regularly interspaced short palindromic repeats (CRISPR) and programmable …

Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the
CEP290 gene,. We developed EDIT-101, a candidate genome-editing therapeutic, to …

Leber congenital amaurosis (LCA) is a group of monogenic inherited retinal degenerations
that typically show early onset and severe visual dysfunction. In addition, there is a natural …

Leber congenital amaurosis (LCA) is an inherited retinal dystrophy that causes childhood
blindness. Photoreceptors are especially sensitive to an intronic mutation in the cilia-related …

As the most common subtype of Leber congenital amaurosis (LCA), LCA10 is a severe
retinal dystrophy caused by mutations in the CEP290 gene. The most frequent mutation …

Leber congenital amaurosis (LCA) is a severe disorder resulting in visual impairment
usually starting in the first year of life. The most frequent genetic cause of LCA is an intronic …

Background CEP290-associated inherited retinal degeneration causes severe early-onset
vision loss due to pathogenic variants in CEP290. EDIT-101 is a clustered regularly …

Purpose: To describe the retinal phenotype of pediatric patients with mutations in the retinol
dehydrogenase 12 (RDH12) gene. Methods: Twenty-one patients from 14 families (ages 2 …

Photoreceptors are polarized neurons, with very specific subcellular compartmentalization
and unique requirements for protein expression and trafficking. Each photoreceptor contains …

Purpose: To determine efficacy outcome measures for clinical trials of Leber congenital
amaurosis (LCA) associated with a common intronic mutation in the CEP290 gene …