Home » Gene Therapy for Leber Congenital Amaurosis
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.
- Promising Results in Phase 1 Gene Therapy Trial for Blinding Disease, September 2008.
- 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
- 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:
