Foundation News » Retinitis Pigmentosa
Foundation Receives $300K Grant for Recessive RP, LCA Gene Therapy Research
August 29, 2011 - The Foundation Fighting Blindness announces a $300,000 grant from the M. House Family Fund to support gene therapy research related to autosomal recessive retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). The funding will span three years to help researchers develop an AAV gene therapy treatment that can safely and effectively deliver a healthy gene to the inner cells in the retina that are affected by variations in the CRB1 gene.
Defects in the CRB1 gene account for approximately 3-4 percent of RP cases and 10-15 percent of LCA cases, which is an early onset form of RP. Dr. Jan Wijnholds of The Netherlands Institute for Neurosciences is hopeful his work will lead to future gene therapy clinical trials for these diseases.
The M. House Family Fund is in the name of Michael House, a longtime Foundation supporter who has four children affected with recessive RP despite no previous family history of the disease. A businessman in commercial real estate and well-known thoroughbred racehorse breeder, he was honored with the Foundation’s Visionary Award at the 2010 San Diego Dining in the Dark.
“Michael House has passionately supported the Foundation’s fight against blindness for over 30 years with the hope of breakthrough treatments for his children and the millions of others affected with vision-robbing diseases,” said Bill Schmidt, CEO, Foundation Fighting Blindness. “This generous grant will help us advance promising gene therapy treatments from the lab into the clinic, to hopefully save and restore sight.”
Recent Retinitis Pigmentosa Articles
- Bone Marrow Stem Cells in Clinical Trial for Retinal Diseases
- GenSight Receives $41.3 Million for Ocular Gene Therapy Development
- Stem Cell Pioneers Creating Retinal Patch to Restore Vision
- XLRS Natural History Study Beginning in Portland, Oregon
- Six Emerging Retinal Therapies Receive $3.1 Million in Foundation Funding