Seven researchers from the U.S. and the U.K. have received the Champalimaud Vision Award for their work developing a gene therapy treatment for Leber congenital amaurosis, a rare form of childhood blindness. The researchers will share the €1 million ($1.15 million) prize.
The award recipients include a researcher who had traced the cause of the disease to a mutated gene, and three teams of cooperating researchers who developed a technique for replacing the gene. They then delivered healthy genes into cells within the eye using engineered harmless viruses. After undergoing this treatment, children and adults had restored vision.
“This is the first, and still only, example of successful gene therapy in humans that corrects an inherited genetic defect, and is therefore a milestone in medical therapeutics,” said Alfred Sommer, MD, chairman of the award jury and dean emeritus at Johns Hopkins University Bloomberg School of Public Health in Baltimore.
The Champalimaud Foundation, which has granted the Vision Award since 2006, was established to advance biomedical research and clinical care, particularly in the fields of neuroscience and oncology.