Brian Kaspar

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....

Injections of genes for the muscle-growth protein follistatin strengthened leg muscles in monkeys

posted on November 12, 2009 - 10:00pm
Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength with no adverse effects, say researchers at Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University. The findings could have implications for people with muscular dystrophies and other...

Neurotrophin 3 genes strengthened mice with a disease resembling CMT1A

posted on May 18, 2009 - 5:29pm
Zarife Sahenk at Nationwide Children's Hospital and Ohio State University in Columbus, and colleagues, found mice with a disease resembling type 1A Charcot-Marie-Tooth disease (CMT) benefited from a transfer of genes for the neurotrophin 3 protein. CMT1A is caused by a duplication of the PMP22 gene. Jerry Mendell, who has received many MDA research grants and co-directs the MDA clinic at...
posted on May 1, 2008 - 5:00pm
QUEST Vol. 15, No. 3
Three sets of laboratory experiments investigating the effects of interfering with myostatin, a protein that limits muscle growth, have shown that this approach may have to be individualized with respect to different types and stages of muscular dystrophy, and that some myostatin suppression strategies may be better than others.