After three decades of hopes tempered by setbacks, gene therapy is no longer the future of medicine, but is part of the present-day clinical treatment toolkit. The Jan. 12 issue of the journal Science provides an in-depth and timely review of the key developments that have led to several successful gene therapy treatments for patients with serious medical conditions, including several inherited neurodegenerative disorders.
Read the NIH summary: The coming of age of gene therapy: A review of the past and path forward