Due to improved phenotyping and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …

Leber congenital amaurosis (LCA) is the most severe retinal dystrophy causing blindness or
severe visual impairment before the age of 1 year. Linkage analysis, homozygosity mapping …

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

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 …

CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease
resulting in childhood blindness. Sepofarsen is an RNA antisense oligonucleotide targeting …

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 …

Retinal neurodegeneration associated with the dysfunction or death of photoreceptors is a
major cause of incurable vision loss. Tremendous progress has been made over the last two …

Ciliopathies are an emerging group of disorders, caused by mutations in ciliary genes. One
of the most intriguing disease genes associated with ciliopathies is CEP290, in which …

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 …