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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
- 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
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