How a vision-restoring gene therapy proved that we can treat inherited diseases
A gene therapy called Luxturna, developed by Katherine High, Jean Bennett, and Albert Maguire, has been shown to restore vision in patients with Leber's congenital amaurosis, a rare inherited eye disease. The therapy, approved by the FDA in 2017, has treated at least 500 people in the U.S. and has been shown to increase visual sensitivity more than 40,000-fold.
Katherine High, Jean Bennett, and Albert Maguire developed Luxturna, a gene therapy that treats Leber's congenital amaurosis (LCA), a rare inherited eye disease causing childhood blindness. The therapy targets a faulty gene in retinal cells and delivers functional genetic instructions. In clinical trials, Luxturna restored vision in patients, increasing visual sensitivity over 40,000-fold. The FDA approved Luxturna in 2017, and at least 500 people in the U.S. have received the treatment. The developers were awarded the 2026 Breakthrough Prize for their work.
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