FDA Approves Gene Therapy for Leber's Congenital Amaurosis
Автор: Penn Medicine
Загружено: 2017-12-19
Просмотров: 9214
Just a few years ago, Penn Medicine's Jean Bennett, MD, PhD, detailed the hopes that recently came to fruition when the FDA approved gene therapy for the treatment of a rare, inherited form of retinal blindness.
In a historic move, the U.S. Food and Drug Administration (FDA) has approved a gene therapy initially developed by researchers at the University of Pennsylvania and Children’s Hospital of Philadelphia for the treatment of a rare, inherited form of retinal blindness. The decision marks the nation’s first gene therapy approved for the treatment of a genetic disease and the first in which a new, corrective gene is injected directly into a patient.
A perfect example of Penn’s One Health initiative, early work for the therapy approved today took place in dogs with the same condition. Just a few years ago, Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania and Penn’s Scheie Eye Institute, detailed her hopes for the therapy that today came to fruition.
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