by Children’s Medical Research Institute – MedicalXpress –
Researchers at the Eye Genetics Research Unit at Children’s Medical Research Institute (CMRI) are the first in the world to use stem cells to study one of the genetic causes of Leber Congenital Amaurosis (LCA)—a rare condition that causes severe vision loss in babies and young children. Their findings suggest that gene therapy could soon help prevent blindness in affected kids.
The study by Dr. To Ha Loi and colleagues, published in Stem Cell Reports, focused on a gene called RPGRIP1, which is important for developing and maintaining the eye’s photoreceptor cells—the light-sensing cells that allow us to see. When this gene is faulty, children can develop severe and currently incurable retinal disease.
