"The child, who had previously been legally blind from the progressive condition, was one of the first patients to regain vision from a gene therapy High helped develop. It was the first time he had ever seen snow fall."
"LCA affects thousands of people globally and is responsible for 20 percent of childhood blindness. People with LCA are born with very poor vision, which slowly worsens over time."
"By targeting a gene central to the molecular dysfunction and creating a novel system to deliver function, Luxturna can help reverse some inherited retinal diseases, including a type of LCA."
Katherine High recalls a profound moment when a 10-year-old boy, previously blind from Leber's congenital amaurosis, saw snow for the first time after receiving Luxturna. This gene therapy, developed by High, Jean Bennett, and Albert Maguire, targets a faulty gene responsible for the disease. LCA affects thousands globally and leads to progressive vision loss, often resulting in blindness by age 20. Luxturna offers hope by reversing some effects of inherited retinal diseases, significantly impacting patients' lives.
Read at www.scientificamerican.com
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