Children born blind from a devastating retinal condition called Leber congenital amaurosis (LCA) may soon be able to see thanks to an innovative gene replacement therapy that has just moved into a clinical trial.
The study, funded in part by the Foundation Fighting Blindness, is being conducted by University College of London and Moorfields Eye Hospital in London. Two additional Foundation-funded clinical trials are scheduled to begin soon at the University of Pennsylvania and the Children’s Hospital of Philadelphia.
Over the past 15 years, the Foundation funded the preclinical investigations that are making these gene replacement studies possible. One world-renowned research project gave sight to a Briard dog named Lancelot and 50 of his relatives.
“This is one of the most important milestones ever reached in retinal research, because it offers the promise of giving vision to children who otherwise would never see,” says Bill Schmidt, Chief Executive Officer, Foundation Fighting Blindness. “We are thrilled to be at this hopeful point.”
“Not only does gene replacement hold promise for LCA, the same approach may be used to treat a variety of other retinal diseases including retinitis pigmentosa, Stargardt disease, Usher syndrome, and age-related macular degeneration,” says Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. “The success of these studies in the U.S. and the U.K. can pave the way for many more treatments to save and restore vision in millions of people around the world.”
People affected by LCA are born with a genetic variation that causes blindness or severe vision loss at birth. Investigators have thus far identified 14 genes that can cause LCA. The current and forthcoming human studies involve delivery of a healthy gene to the retinas of people with LCA caused by variations in the gene RPE65.
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