SMN Gene Therapy (Genzyme Sanofi) – SMA News Today
SMA News Today |
SMN Gene Therapy (Genzyme Sanofi)
SMA News Today Genzyme's current investigational program is focused on the delivery of the AAV9-SMN gene into cerebrospinal fluid. Work in mouse models of SMA has shown that delivery of AAV9-SMN1 into the cerebrospinal fluid results in a transfer of the gene to the … |
