Researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia have used gene therapy to safely improve vision in five children and seven adults with a rare form of congenital blindness
Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.
Continue reading… “One Shot Of Gene Therapy, And Children With Congenital Blindness Can Now See”