Study: Gene Therapy Efficacious for Type of Retinal Disease

Gene therapy shows safety and efficacy for Leber congenital amaurosis, a retina disease that is an inherited form of blindness, according to a Penn Medicine news release.

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In studies over the past three years, researchers at the Perelman School of Medicine at the University of Pennsylvania and the Scheie Eye Institute have shown the safety and efficacy of the new treatment in five groups of patients representing many age groups.

 

The new research, published online this week in the Archives of Ophthalmology, describes how patients received an injection of genes into the retina of one eye for the type of LCA caused by a mutation in the RPE65 gene.

 

Related Articles on Retinal Diseases:

Ophthalmologist-CEO Lloyd Hildebrand Shows Clinicians’ Key Role in Innovation

Researchers Find New Genetic Cause for Retinitis Pigmentosa

Study: Patients With Retinal Disease More Likely to Suffer Strokes


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