Seven years after the FDA approved Luxturna, scientists have yet to bring another congenital blindness treatment to the market.
A team of researchers from the Perelman School of Medicine at the University of Pennsylvania aims to change this. The group recently published a study in The Lancet documenting their success using gene therapy to treat an inherited retinal blindness that affects as many as 100,000 people globally.
Targeting people with a variant of Leber congenital amaurosis (known as LCA1) — a kind of vision loss not easily corrected with glasses — the researchers cut three incisions near the iris of 15 people and injected them with ATSN-101, the experimental gene therapy. Participants’ vision on average improved and lasted for the duration of the observation period (12 months), with no serious side effects observed.
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