CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Published in Volume 120, Issue 4 (April 1, 2010)
J Clin Invest. 2010;120(4):1253–1264. doi:10.1172/JCI41615.

Marco A. Passini, Jie Bu, Eric M. Roskelley, Amy M. Richards, S. Pablo Sardi, Catherine R. O’Riordan, Katherine W. Klinger, Lamya S. Shihabuddin and Seng H. Cheng

Genzyme Corporation, Framingham, Massachusetts.