Foundation News » Retinitis Pigmentosa
International Team Planning New Gene Therapy Clinical Trial for RP
Researchers from the United States, China, and Saudi Arabia are planning to launch a gene therapy clinical trial in 2010 for a recessive form of retinitis pigmentosa caused by variations in the gene MERTK.
Taking place in Saudi Arabia, the trial will be led by Foundation Fighting Blindness-funded investigator Khang Zhang, M.D., Ph.D., of the University of California, San Diego, and Fowzan Alkuraya, M.D., of the King Faisal Specialist Hospital and Research Center in Saudi Arabia. William Hauswirth, Ph.D., a Foundation-funded gene therapy development expert from the University of Florida, is also on the study team.
Investigators in this study will be using the same type of gene delivery mechanism — an adeno-associated virus or AAV — currently being utilized in all three gene therapy clinical trials underway for Leber congenital amaurosis, a severe form of retinitis pigmentosa that causes children to be born blind or with severe vision loss.
MERTK is a gene involved in the photoreceptor regeneration process that occurs nightly in the human retina. Every night, the tips of photoreceptors are essentially “chewed off” and disposed of. Subsequently, the tips regenerate. When the MERTK gene is defective, the disposal process doesn’t work properly, and debris and waste products accumulate causing photoreceptor death and vision loss.
“We at the Foundation are very pleased to see clinical trials of gene therapy for more retinal diseases emerging,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “While we are still at a relatively early stage in these clinical studies, the momentum moving forward is impressive. The progress and expansion provides real hope for people losing vision to these devastating conditions.”
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