A new gene therapy for one of the most common forms of congenital blindness was safe and improved patients’ vision, according to initial data from a clinical trial led by researchers at the University of Pennsylvania.
The therapy delivers working copies of the gene GUCY2D to the eyes of patients who have severe vision impairments caused by mutations in the gene. Each of the first three treated patients experienced improvement in some aspects of vision, without serious side effects, according to the new study, published in the journal iScience.
Credit: Alexander Sumaroka and Alexandra V. Garafalo
Research paper: Safety and Improved Efficacy Signals following Gene Therapy in Childhood Blindness Caused by GUCY2D Mutations [ Ссылка ]00377-1
News Source: [ Ссылка ]
Playlist for Technology News Videos - [ Ссылка ]
Health - [ Ссылка ]
