First Gene Therapy for Inherited Retinal Disease
Pioneering research at Penn Medicine has led to a first-of-its-kind therapy to treat a genetic disease. This FDA-approved gene therapy reverses blindness in children and adults born with a rare form of inherited retinal disease called Leber congenital amaurosis (LCA). LCA, itself a type of early onset retinitis pigmentosa, affects an estimated one out of every 30,000-81,000 newborns and causes progressive deterioration of the poor vision found in early childhood.
The treatment, developed at the University of Pennsylvania with clinical trials carried out at Children’s Hospital of Philadelphia and the University of Iowa, is the first time gene therapy is used to treat an inherited disease and could lead to treatments of other currently untreatable conditions.
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Pioneers in Gene Therapy
The FDA approval for the treatment of inherited retinal disease marks the second groundbreaking advance in gene therapy made at Penn Medicine within the year. We made history in August 2017 when the FDA approved a personalized cell therapy for advanced leukemia, the first-ever for cancer cell and gene therapy.
"This is a really, really, exciting time. This could revolutionize treatments not only for different blinding diseases but also for other genetic and acquired diseases for which we have no treatment."
This latest development in gene therapy is the result of more than two decades of painstaking genetic research by Jean Bennett, MD, PhD, and Albert M. Maguire, M.D., both Professors of Ophthalmology and Co-directors of the Center for Advanced Retinal and Ocular Therapeutics at Penn Medicine. They and their colleagues achieved the FDA approval seventeen years after they restored sight to a blind dog named Lancelot, who had a canine version of LCA.
Dr. Bennett’s and Dr. Maguire’s research not only provides hope for patients with LCA, but for everyone with inherited blindness. There are at least 265 different genes in which mutations can cause blindness; breakthroughs in gene therapy are allowing scientists to develop treatments for diseases that were previously untreatable, giving hope to millions.