Leber Congenital Amaurosis (LCA)

The Disease

  • Most common cause of inherited childhood blindness
  • Incidence of two to three per 100,000 live births worldwide
  • Symptoms appear within the first year of life and include significant vision loss, rapid involuntary movements of the eyes and absence of measurable electroretinogram recordings due to progressive loss of photoreceptor cells, ultimately leading to blindness
  • LCA10 most common form of disorder, accounting for 20-30% of all LCA subtypes and is caused by a mutation within the CEP290 gene
  • Currently no treatment options for LCA10

The Editas Approach

Therapeutic approaches aimed at restoring function of remaining photoreceptor cells could arrest further loss of vision for patients with LCA10. Our approach is designed to eliminate a mutation in the CEP290 gene by cutting out that nucleotide and surrounding DNA, thus restoring normal protein expression and function of remaining photoreceptor cells.