Gene Therapy for Leber Congenital Amaurosis

Leber congenital amaurosis (LCA) is an inherited retinal disease that causes 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.

Interactive Timeline
The Research Road: Gene Therapy for Leber Congenital Amaurosis

View the text-only timeline.

Study Results

• Gene Therapy One Year Later: Patients Healthy and Maintain Early Visual Improvement
• Promising Results in Phase 1 Gene Therapy Trial for Blinding Disease, September 2008.
• Selected Bibliography on LCA and RPE65

Background Information

• Backgrounder: Human Gene Transfer Therapy for One Form of Childhood Blindness
• The Gene Therapy Journey: From Bench to Bedside
• 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

Other Useful Links

• NEI Photos, Images, and Videos: Leber congenital amaurosis
• ClinicalTrials.gov: Phase I Trial of Gene Vector to Patients with Retinal Disease Due to RPE65 Mutations
• NEI Molecular Mechanisms Section, Laboratory of Retinal Cell and Molecular Biology
• NEI Ophthalmic Genetics and Visual Function Branch
• MEDLINEPlus: Genes and Gene Therapy