Gene therapy researcher and MDA grantee Jerry Mendell discusses autoimmunity to dystrophin protein in Duchenne muscular dystrophy
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
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
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
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.
Emanuela Gussoni, Ph.D.
AffiliationChildren’s Hospital and Harvard University, Boston
StrategyIsolating side population cells from muscle tissue