Gene Therapy Extends Survival in an Animal Model of Spinal Muscular Atrophy

May 22, 2014 - NEW ROCHELLE, N.Y. -- To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy , researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA.



via New Rochelle Newswire http://ift.tt/TyfyMP