Research Articles - Other Retinal Diseases
At first blush, completely shutting down both copies of a gene might not seem like the best way to treat an inherited retinal disease. That’s because genes and the proteins they express are thought to be essential to the health and well-being of all cells in the body.
To advance the research and development of inherited retinal-disease therapies, the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and Sanofi, a global pharmaceutical company headquartered in France, have entered into a two-year agreement to regularly exchange scientific information.
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.
When a person is diagnosed with an inherited retinal condition, identifying his or her disease-causing gene mutation can often be beneficial. It enables a physician to make a definitive diagnosis and determine which family members may be at risk for the condition. Also, identification of the mutation is necessary for participation in clinical trials of emerging gene therapies. However, more than 200 genes have been linked to retinal diseases, which often makes the search difficult.
Eighteen of the world’s leading retinal researchers convened in Las Vegas November 18-20 to present their progress in advancing clinically focused research made possible by the Foundation’s Translational Research Acceleration Program (TRAP). Since 2008, $70 million has been allocated to the program to move promising retinal-disease treatments into human studies.
Two French research institutions — the Institut de la Vision and Institut Langevin — will receive nearly $16.5 million from the European Research Council (ERC) to advance state-of-the-art imaging technologies for gaining a better understanding of retinal diseases and developing sight-saving therapies.
The Foundation Fighting Blindness is providing $600,000 to a consortium of Israeli scientists working toward the ambitious goal of recruiting and genetically screening every person in Israel with an inherited retinal degenerative disease.