Re-dosable gene therapy

Leber’s congenital amaurosis is the most common genetic cause of childhood blindness. This retinal dystrophy causes blindness or severe visual impairment beginning at birth, or in the first few months of life. LCA10 is a subtype of LCA, and is caused by mutations in the CEP290 gene. LCA10 affects about two to three per 100,000 people worldwide.

Stargardt disease, also called Stargardt macular dystrophy or juvenile macular degeneration, is an inherited disorder of the retina that causes vision loss during childhood or adolescence, and is caused by mutations in a gene called ABCA4. According to the National Eye Institute at the National Institutes of Health, an estimated one in 8,000 to 10,000 people suffer from Stargardt disease worldwide.