Research Articles - Other Retinal Diseases
In support of the fight to eradicate blindness, Foundation Fighting Blindness Chairman and Co-Founder Gordon Gund and his family announced a matching gift challenge they hope will result in millions of dollars in funds to accelerate pre-clinical, translational and clinical research for retinal degenerative diseases.
For the first time, researchers have used induced pluripotent stem cells — stem cells derived from the reprogramming of mature human cells — to create a three-dimensional retina with functional photoreceptors, the cells that make vision possible. The new model will enable scientists to better understand retinal development and function, and provide a powerful platform for creating and testing future therapies. Results from the study, led by M.
Thanks to a Foundation Fighting Blindness collaboration with GE Lighting, people accessing services at the Spectrios Institute for Low Vision in Wheaton, Illinois, now have significant lighting improvements at their disposal. A “re-lamping” effort, courtesy of GE Lighting, replaced much of the institute’s existing lighting with energy-efficient products and fixtures throughout the facility’s training, exam and counseling rooms, among other areas.
Three years after it began, the landmark Phase I gene therapy clinical trial for people with Leber congenital amaurosis (LCA) at the Universities of Pennsylvania and Florida continues to go very well. Overall safety and vision improvements have been sustained. All 15 participants in the study, ranging in age from 11 to 30, have demonstrated vision improvement to varying degrees, including increases in visual field, night vision and mobility. Improvements in visual acuity only
A Foundation-funded University of Wisconsin research team led by Dr. David Gamm has achieved a significant milestone in developing stem cell treatments for saving and restoring vision in people with retinal degenerations. For the first time, the scientists used stem cells derived from blood to produce multiple layers of cells that resemble retinal tissue. Most important, these cellular structures have some of the characteristics considered necessary for vision.
Contrary to its name, Best disease is not a condition anyone wants. Named after the German ophthalmologist Friedrich Best more than a century ago, it is an inherited form of macular degeneration that can cause significant and progressive loss of central vision in children and adults.
In a research paper recently published in the Proceedings of the National Academy of Sciences, investigators from the University College of London (UCL) reported on their successful integration of stem cell transplants in six mouse models of retinal disease, including those for retinitis pigmentosa (RP), Leber congenital amaurosis and congenital stationary night blindness (CSNB).
The Children’s Hospital of Philadelphia (CHOP) is investing $50 million in Spark Therapeutics, its newly formed spinoff company, to lead its landmark gene-therapy clinical trial for Leber congenital amaurosis (LCA, RPE65 mutations), an inherited form of blindness affecting children, and hemophilia B, an inherited blood-clotting disorder. The company also plans to advance additional gene therapies, including those for other
Drs. Albert Maguire, Katherine High and
Jean Bennett of CHOP
A Foundation-funded research team led by investigators from the University of Pennsylvania has reported that its emerging gene therapy for Best disease, a form of inherited macular degeneration, has cleared all the vision-robbing lesions in canines with the condition. Success in the study moves scientists closer to a human study of the treatment. Dr. Karina Guziewicz in collaboration with