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Diagnosed with Leber congenital amaurosis as a baby, Hannah was on a path to going blind until gene therapy at Children’s Hospital restored her sight.
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A groundbreaking clinical trial of gene therapy for a form of congenital blindness, co-sponsored by CHOP, earned award for distinguished clinical research.
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Gene therapy for congenital blindness has taken another step forward, as researchers further improved vision in three adult patients previously treated in one eye.
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Researchers at The Children's Hospital of Philadelphia show that a second dose of gene therapy for inherited blindness is safe in animals.
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The Children's Hospital of Philadelphia was lauded for significant contributions to medical science in 2009 for dispelling myths that vaccines cause autism, and developing gene therapy to treat blindness.
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A CHOP/Penn Medicine study used gene therapy to safely improve vision in five children and seven adults with a Leber’s congenital amaurosis, a form of blindness.