Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice
Niemir N, Rouvière L, Besse A, Vanier MT, Dmytrus J, Marais T, Astord S, Puech JP, Panasyuk, Cooper JD, Barkats M, Caillaud C.
Adeno-associated virus (AAV) gene therapy has shown much promise for inherited diseases. Most recently, non-invasive delivery routes such as intravenous injection of AAV have been evaluated in a number of inherited diseases. In fact, clinical trials are underway utilizing intravenous AAV gene therapy for MPSIIIA and Spinal Muscular Atrophy. In January of 2018 a paper was published detailing a study evaluating intravenous AAV gene therapy in a mouse model of Sandhoff disease.