Dongsheng Duan

MDA-supported investigators found that intramuscular injections of microdystrophin genes improved muscle health in dystrophin-deficient dogs, a response not previously seen in large animals or humans

posted on January 15, 2013 - 2:34pm
For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.

A follistatin gene therapy compound gains orphan drug status; potential improvements are reported for utrophin-based therapies, exon skipping and stem cell transplantation

posted on December 27, 2012 - 5:00am
Update (Jan. 23, 2013): The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri. Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.
posted on March 31, 2011 - 11:40am
QUEST Vol. 18, No. 2
Dongsheng Duan's interest in gene therapy to treat diseases goes back a long way, although his initial focus wasn't muscular dystrophy. "I came to the muscle field by accident," says Duan, an MDA research grantee and professor of molecular microbiology and immunology at the University of Missouri at Columbia. "It was not something I intended to do when I was young."

Researcher Dongsheng Duan says adding a piece to miniaturized dystrophin genes makes them more effective

posted on March 15, 2009 - 9:00pm
Displacement of a protein called neuronal nitric oxide synthase (nNOS) from the membrane that surrounds each skeletal muscle fiber appears to be a much more important contributor to exercise intolerance and even cardiac degeneration in some forms of muscular dystrophy than previously recognized.

Promising minigene only partially normalizes heart muscle

posted on January 21, 2009 - 11:14am
A promising “minidystrophin gene” that restores normal muscle force to skeletal and diaphragm muscles in mice with a disease resembling Duchenne muscular dystrophy (DMD) seems to be only partially effective at restoring strength and function to heart muscles.

Can sarcoglycan proteins help compensate for dystrophin's absence?

posted on January 18, 2009 - 10:55am
Delivering the gene for the missing dystrophin protein to the muscles of children and young men with Duchenne muscular dystrophy (DMD) is one of several therapeutic avenues under intense investigation in this disease. However, the strategy has technical drawbacks, and some experts worry that introduction of a previously absent protein could provoke a dangerous immune response.
posted on September 1, 2008 - 10:07am
QUEST Vol. 15, No. 5
Real progress is being made in supplying functional dystrophin genes to treat Duchenne muscular dystrophy (DMD), a disease in which mutated dystrophin genes keep this critical protein from being produced in muscle fibers.
posted on March 1, 2007 - 2:34pm
QUEST Vol. 14, No. 2
An MDA-supported research team, testing gene transfer in dogs using adeno-associated viral (AAV) shells (vectors), noted an unwanted immune response, a factor they recommend be considered in human trials. The response occurred even when the vectors weren’t carrying any genes. Until now, AAV transporters have been thought not to provoke much of an immune response.