The rare eye disease Leber Congenital Amaurosis, caused by a defective gene, has a new genetic treatment. This is the first time the US Food and Drug Administration has approved gene therapy for an inherited disease.
The RPE65 gene provides a blueprint for a protein that makes light receptors work properly. Individuals with a defective RPE65 gene eventually go blind. Eye doctors may diagnose them with Leber Continue reading “Leber Congenital Amaurosis Gene Therapy Approved by FDA”