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A new study marks a significant advance in developing a gene therapy for X-linked retinitis pigmentosa, a hereditary disease that leads to severe sight loss in young males.
Researchers at Bascom Palmer Eye Institute, the Department of Ophthalmology at the University of Miami Miller School of Medicine, took part in the international multi-center study and are actively participating in further clinical trials. It's the latest published study for the Bascom Palmer specialists who have performed nearly 100 gene therapy surgeries to date for several types of inherited retinal disorders.

"X-linked retinitis pigmentosa related to the GTPase regulator (RPGR) gene is one of the most common and severe types of this disease," said Byron L. Lam, M.D., the Robert Z. & Nancy J. Greene Professor of Ophthalmology, and Bascom Palmer's principal investigator. "This gene therapy study offers hope for patients with this currently untreatable blinding disease."

Read more: https://lnkd.in/gy5uvaW