Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …

Leber congenital amaurosis (LCA) and early-onset severe retinal dystrophy (EOSRD) are
both genetically and phenotypically heterogeneous, and characterised clinically by severe …

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 …

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 …

Leber congenital amaurosis type 10 (LCA10) is a severe inherited retinal dystrophy
associated with mutations in CEP290. The deep intronic c. 2991+ 1655A> G mutation in …

Patient-derived induced pluripotent stem cells (iPSCs) hold great promise for autologous
cell replacement. However, for many inherited diseases, treatment will likely require genetic …

Background: Gene therapy cannot be yet considered a far perspective, but a tangible
therapeutic option in the field of retinal diseases. Although still confined in experimental …