immune response to dystrophin

Findings on the immune response to dystrophin in Duchenne and Becker MD, treatment of cardiomyopathy in DMD, and eteplirsen in DMD were presented

posted on April 26, 2012 - 4:41pm
Update (June 1, 2012) ­—  The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy."

Gene therapy researcher and MDA grantee Jerry Mendell discusses autoimmunity to dystrophin protein in Duchenne muscular dystrophy

posted on January 13, 2011 - 3:57pm
A January 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, probes a subject that's been on the minds of many researchers, doctors and families: autoimmunity (self-immunity) in Duchenne muscular dystrophy (DMD).

Future gene therapy trials in Duchenne muscular dystrophy may need to assess participants' immunity to dystrophin, recent findings show

posted on October 6, 2010 - 2:00pm
Editor's note: This story was updated April 27, 2012. Immune-system rejection of newly synthesized dystrophin protein occurred in at least some of the boys with Duchenne muscular dystrophy (DMD) who participated in a safety trial of dystrophin gene therapy. The finding was a partial surprise to researchers and demonstrates the value of small, phase 1 clinical trials.

An unexpected immune response to dystrophin has researchers proceeding cautiously; the finding changes how future gene therapy trials will be conducted

posted on April 21, 2010 - 10:59am
Unwanted responses by the immune system to dystrophin have been seen in a small, MDA-supported clinical trial of gene therapy for Duchenne muscular dystrophy (DMD) — an unexpected finding, investigators say. Rather than a setback, the finding is “the beginning of a new way of thinking” about gene therapy, said Jerry Mendell, director of the Center for Gene Therapy at Nationwide Children's and a...

The drug gentamicin increased dystrophin levels in boys with DMD due to a nonsense mutation, but didn't improve strength at the doses used

posted on April 14, 2010 - 3:34am
Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto. Levels of the needed dystrophin protein increased in six out of 12 participants who received the drug for six months. No functional improvements were seen.
posted on November 1, 2005 - 9:53am
QUEST Vol. 12, No. 6
Emanuela Gussoni, Ph.D. AffiliationChildren’s Hospital and Harvard University, Boston StrategyIsolating side population cells from muscle tissue StatusLaboratory experiments