To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA. A new study demonstrating that enough copies of the SMN1 gene can be delivered to the spinal cord motor neurons to extend the survival of the treated animals is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc.., publishers. The article is available free on the Human Gene Therapy website.
Fuente : http://www.alphagalileo.org/ViewItem.aspx?ItemId=1...
Fuente : http://www.alphagalileo.org/ViewItem.aspx?ItemId=1...