Research Articles - Retinitis Pigmentosa
The gene CRB1 is a key target in the fight against inherited retinal diseases such as retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) because mutations in it lead to significant, often devastating vision loss. Approximately 4,000 people in the United States are affected by the mutation, as many as 80,000 worldwide.
- A few weeks ago, 15 of the world’s leading retinal researchers, all funded by FFB’s Translational Research Acceleration Program (TRAP), gathered for an annual meeting in Las Vegas to discuss their progress in developing vision-saving treatments. Most are poised to move into clinical trials within the next two to five years.“It’s an exciting time in translational research for retinal diseases,” says Patricia Zilliox, Ph.D., chief drug development officer at the
- November 17, 2014 - The Foundation Fighting Blindness (FFB) recently updated its website by adding Spanish language translations of some of its resource materials to better serve patients and others interested in learning about retinal diseases.Those wishing to view the materials in Spanish may click on the link En Espanol in the resources section of the Foundation’s website.
- Jan. 15, 2015 – Six months after the launch of a clinical trial for a bone marrow stem-cell treatment targeting retinal disorders, researchers are reporting encouraging preliminary findings.The first six patients enrolled in the on-going clinical trial, taking place at the University of California Davis Eye Center, tolerated the procedure well without any serious adverse events.
If you burn yourself, bump your head, get bitten by an insect or twist an ankle, the injured area becomes inflamed. While the swelling and redness associated with inflammation can be uncomfortable and disconcerting, they are actually a good thing – your body’s attempt to protect, repair and heal itself. At the site of the injury, your body is increasing blood flow and engaging immune-system proteins and cells to remove damaged tissue, fight infection and initiate healing. It’s a complex, yet elegant, process.
Photoreceptors are cells in the outer retina that provide vision, and once they’re gone due to an advanced retinal degenerative disease like retinitis pigmentosa (RP), a person’s eyesight is lost as well.
Three young adults with virtually no vision can now read several lines on an eye chart and see better in dimly lit settings thanks to an innovative gene therapy aiming to reverse blindness in a severe form of retinitis pigmentosa known as Leber congenital amaurosis or LCA. One person was even able to better navigate an obstacle course several weeks after receiving the therapy.
Two emerging gene-therapy development companies — Philadelphia-based Spark Therapeutics and Ireland-based Genable Technologies — have entered into a strategic partnership to accelerate the advancement of a gene therapy for people with autosomal dominant retinitis pigmentosa (adRP) caused by mutations in the gene RHO.