US and UK Scientists Win Million-Dollar Prize for Gene Therapy to Cure Childhood Blindness
4th September 2018
Babies born with LCA have poor eyesight and are extra sensitive to light. In most cases, people with the “rare genetic eye disorder” go completely blind by age 40, according to a press release from the University of Pennsylvania School of Medicine.
LCA is caused by a mutated gene. The scientists figured out a way to transfer a corrected version of the gene to patients via innocuous viruses. They received the U.S. Food and Drug Administration’s first approval of a gene therapy for a congenital disorder in December 2017, according to the press release.