gene transfer therapy

A phase 1 gene transfer trial in nine babies with type 1 spinal muscular atrophy has opened at Nationwide Children's Hospital in Columbus, Ohio

posted on May 6, 2014 - 9:33am
A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.

A look at how today's gene therapy research for DMD is overcoming obstacles

posted on January 9, 2014 - 9:29am
Quest Winter 2014
The year was 1986, and the Duchenne muscular dystrophy (DMD) community was more excited than it had been for decades. A group of MDA-supported researchers had identified the genetic cause of the disorder — any of a number of different flaws (“mutations”) in the gene for a newly identified muscle protein, one that would come to be called dystrophin. 

In a podcast from Nationwide Children’s Hospital, longtime MDA grantee Jeffrey Chamberlain describes advances in gene transfer therapy and stem cell therapy for Duchenne muscular dystrophy

posted on July 31, 2013 - 3:31pm
In a July 2013 podcast from Nationwide Children’s Hospital in Columbus, Ohio, longtime MDA grantee Jeffrey Chamberlain discusses recent advances in the development of gene therapy (gene transfer) and stem cell therapy (transplantation) for Duchenne muscular dystrophy (DMD), the results of which may apply to other types of muscular dy

Wake Forest researcher receives $369,365 for preclinical testing of gene transfer therapy for myotubular myopathy

posted on May 26, 2011 - 2:16pm
MDA’s translational research program has announced it is funding research into a potential treatment for the inherited muscle disorder X-linked myotubular myopathy (MTM). The grant of $369,365 grant to Wake Forest University professor Martin Childers will fund a three-year study of myotubularin gene transfer in mice and dogs that have an MTM-like disease.