EaseVision.com – News and gossips of vision care and optical industry

Reported on October 25, an online article in The Lancet revealed some important findings about gene therapy. Researchers from the School of Medicine at University of Pennsylvania, and the Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia have successfully improved vision in 12 subjects using gene therapy. Including 5 children and 7 adults, all of the twelve subjects were bothered by Leber’s congenital amaurosis (LCA), which is a group of inherited blinding diseases that damage light receptors in the retina. 

LCA is incurable by any treatments until now. The result was quite significant for the entire filed of gene therapy, according to co-first author of the study. Participants of the study were legally blind and may be forced to be totally sightless. One of the children could not see the blackboard normally. He had to rely on the large print on an electronic screen and get assistance from the teacher. Researchers injected genes that produce light-sensitive pigments into the back of his eyes. The boy restored his ability to do normal tasks in the classroom and play certain games with his classmates. 

With the help from gene injection, half of the 12 subjects improved their vision enough and they are not legally blind now. Aging from 8 to 11, the five children can now navigate a low-light obstacle course, which is spectacular to the researchers. After the treatment, these children can navigate the course more quickly with fewer errors than before. However, adults performed generally more modestly on the mobility course than children. 

Researchers such as Maguire and Bennett have long been researching on inherited retinal degeneration. This time, those researchers focused on Leber’s congenital amaurosis, which can cause total blindness in one’s twenties or thirties. They used a genetically engineered adeno-associated virus to carry a normal version of the gene named PRE65. This gene is mutated in one form of LCA. And Jeannette cloned the gene in Bennett’s lab.