Gene Therapy For Patients With Childhood Blindness

A new gene therapy for one of the most common forms of congenital blindness was found to be safe and improved patients’ vision. The therapy delivers working copies of GUCY2D to the eyes of patients who have severe vision impairments caused by mutations in the gene.

The researchers found sustained improvements in both day and night vision, even with a relatively low dose of the gene therapy. The GUCY2D gene is one of about 25 different human genes whose mutations cause problems in the retina, leading to severe vision impairment from birth or early childhood. This family of inherited retinal disorders, collectively known as Leber Congenital Amaurosis (LCA), accounts for a considerable portion of blindness in children worldwide.

Even in adults who have lived for decades with this condition, it is often the case that many light-sensing retinal cells remain alive and intact despite their dysfunction. Thus, adding functional copies of GUCY2D via a gene therapy could get those cells working again and restore some vision.

Read more