Gene Therapy

Mass. Eye and Ear Performs First FDA-Approved Gene Therapy Procedure for Inherited Disease

Massachusetts Eye and Ear made medical history this week by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The occasion marks the beginning of a new era in medicine, as it is the first time any FDA-approved gene therapy has been given to a patient for any inherited disease. The treatment, commercially identified as Luxturna, was developed by Spark Therapeutics and approved in
Ophthalmology

NIH Discovery Brings Stem Cell Therapy for Eye Disease Closer to the Clinic

Scientists at the National Eye Institute (NEI), part of the National Institutes of Health, report that tiny tube-like protrusions called primary cilia on cells of the retinal pigment epithelium (RPE)—a layer of cells in the back of the eye—are essential for the survival of the retina’s light-sensing photoreceptors. The discovery has advanced efforts to make stem cell-derived RPE for transplantation into patients with geographic atrophy, otherwise known as dry age-related