Gene Therapy for Leber Congenital Amaurosis

Leber congenital amaurosis (LCA) is an inherited retinal disease that causes with severe visual impairment in infancy or early childhood. Current research on a gene transfer therapy may offer hope to people with a form of this disease.

Retinal pigment epithelial (RPE) cells stained red by RPE65 antibody.

• Phase 1 Clinical Trial Results of Gene Transfer for Leber Congenital Amaurosis Reported September 2008.
• NEI Statement on Gene Transfer for Leber Congenital Amaurosis, April 2008.
• Gene Therapy for Childhood Blindness
• RPE65/LCA Timeline
• NEI Funding of Basic and Preclinical Studies Prior to the LCA Gene Therapy Trial
• NEI Statement on Clinical Trial of Gene Transfer Therapy for Childhood-Onset Blindness, November 2007
• Backgrounder: Gene Transfer Therapy for One Type of Childhood Blindness Studied in Humans
• ClinicalTrials.gov: Phase I Trial of Gene Vector to Patients with Retinal Disease Due to RPE65 Mutations
• Selected Bibliography on LCA and RPE65

Other Useful Links:

• NEI Molecular Mechanisms Section, Laboratory of Retinal Cell and Molecular Biology
• NEI Ophthalmic Genetics and Visual Function Branch
• MEDLINEPlus: Genes and Gene Therapy