The Oscar of Science” has been awarded to the team behind a gene therapy that restores lost vision.
American scientist couple Jean Bennett and Albert Maguire, along with Katherine High, have won the $3 million Breakthrough Prize, an award often referred to as the “Oscars of Science.”
The three researchers were honored for developing the gene therapy Luxturna, a treatment that has significantly restored vision in patients suffering from the rare genetic disorder Leber congenital amaurosis. The disease typically leads to complete blindness by early adulthood.
Luxturna, approved in the United States in 2017, works by delivering a healthy copy of the RPE65 gene into retinal cells. In one clinical trial, a patient who had been blind for years was able to see their child’s face for the first time.
The couple began their research more than 25 years ago at the University of Pennsylvania and even treated two blind dogs along the way, which later became their pets.
At this year’s Breakthrough Prize ceremony, other researchers were also recognized for advances in treatments for diseases such as sickle cell anemia, thalassemia, and for discoveries related to the genetic causes of ALS.
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