Gene Therapy Shows Promise in Congential Blindness Patients
by Angela Mohan on
April 20, 2021 at 11:14 AM
New gene therapy for a congenital blindness was found to be safe and improved patients' vision, according to a clinical trial led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.
This gene therapy delivers copies of GUCY2D gene to the eyes of patients who have severe vision impairments caused by mutations in the gene, according to the study, published in the journal
iScience.
"We found sustained improvements in both day and night vision, even with a relatively low dose of the gene therapy," said study lead author Samuel G. Jacobson, MD, PhD, a professor of Ophthalmology in the Perelman School of Medicine.