Stephen Wilton

First human exon skipping trial in US to begin in March 2010; eligible DMD participants sought

posted on October 28, 2009 - 9:39am
The first human trial in the United States of a treatment strategy known as "exon skipping" for Duchenne muscular dystrophy (DMD) is scheduled to begin in March 2010 at Nationwide Children's Hospital in Columbus, Ohio, one of five elite centers comprising MDA's DMD Clinical Research Network.

Exon skipping proves effective in mice with a severe DMD-like disease

posted on October 23, 2009 - 10:11am
Mice with a severe disease closely resembling human Duchenne muscular dystrophy (DMD) have responded extremely well to a new "exon skipping" compound that targets the specific dystrophin gene error these mice have. Researchers noted "a remarkable prevention of the dystrophic pathology and improvement of the muscle function" in these severely affected mice, which lack both the dystrophin and...
posted on July 1, 2007 - 10:32am
QUEST Vol. 14, No. 4
The underlying cause of Duchenne muscular dystrophy (DMD) can be any of a large number of mutations in the gene for the dystrophin protein. One strategy now being tested for overcoming this problem is gene therapy, the insertion of new dystrophin genes. Another is the transplantation of cells that give rise to muscle.
posted on September 1, 2006 - 3:27pm
QUEST Vol. 13, No. 5
Stephen Wilton Stephen Wilton, an MDA grantee, and colleagues at the University of Western Australia in Perth, with scientists at AVI BioPharma in Corvallis, Ore., report significant progress in two sets of experiments that make use of the technique known as exon skipping.