Christian Lorson

Gene therapy delivered to muscles reaches nerve cells; collaboration aims to advance gene therapy development program; a mouse model represents milder forms of the disease

posted on March 14, 2013 - 4:51pm
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles. Recent research news includes advances in delivery methods for gene therapy treatment of SMA, and creation of a new mouse model that could help scientists better understand and develop treatments for the disease....
posted on September 1, 2006 - 10:36am
QUEST Vol. 13, No. 5
Researchers, including MDA grantees, working on gene therapy for muscle and nerve diseases presented findings and exchanged ideas at the ninth annual meeting of the American Society of Gene Therapy, held in Baltimore in June. Improved cell therapy has promise, drawbacks