Gene therapy restores vision in first-of-its-kind trial
Drug Discovery World
SEPTEMBER 11, 2024
A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. Patients who received the highest dose of the gene therapy saw up to a 10,000-fold improvement in their light sensitivity, were able to read more lines on an eye chart, and improved in their ability to navigate a standardised maze.
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