Research Shows Degenerative Diseases Affecting the Retina May Be Treatable with Gene Therapy

September 27, 2016

(News-Medical) – Researchers have demonstrated the ability to deliver a fully functional copy of the CLN3 gene to stem cells of patients with juvenile NCL, an inherited neurodegenerative disease in which a mutation in the CLN3 gene causes early-onset severe central vision loss. The gene therapy restored production of CLN3 protein in the stem cell-derived retinal neurons, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers