chromosome 5

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....

Mutations in the DYNC1H1 gene cause a rare form of spinal muscular atrophy; plastin 3 contributes to movement problems in SMN-related SMA

posted on May 15, 2012 - 12:29pm
Below are highlights of two recent studies in spinal muscular atrophy (SMA), a disease in which the nerve cells (motor neurons) that control muscles in the spinal cord die, causing progressive weakness in the voluntary muscles.