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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
