FDA Approves Gene Therapy for Leber's Congenital Amaurosis

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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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I was diagnosed with LCA, the CRB1 gene when I was 6 months old. I am 22 now and my vision has always been stable. It only got better after middle school. The doctor told my parents that I would never amount to anything, but I just graduated college with a bachelors of music and vocal performance this past April and it was within 4 years, also, I was the first to graduate college from my high school class, now I teach voice at the florida school for the Deaf and the Blind, which is where I graduated high school in 2017. Hard work is everything, but I could not do it with out God and the support from my family and friends.

EmelyRowan
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I have this condition but haven’t noticed my sight getting worse and I was never warned about that either

autistictechgirl
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is there any treatment for my 1year baby with LCA ?please answer me

mzhdaabdulrahmantofiq
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My son is suffering from Retinitis pigmentosa due to gene prom 1.He has got no vision.Kindly help

jyotsnamathur
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Is there any treatment for my 5 years old son with Leber Congenital Amaurosis (LCA 1)-GUCY2D Gene?

MostafaDelshadi