Researchers review muscular dystrophy therapies

June 22, 2012
Dean Burkin, associate professor of pharmacology at the University of Nevada School of Medicine in Reno is working to develop a therapy for muscular dystrophy. He has co-authored a paper published this week in Science Translational Medicine that examines the potential MG53 protein therapy for Duchenne muscular dystrophy. Credit: Photo courtesy of University of Nevada, Reno

Leading muscular dystrophy researcher Dean Burkin, of the University of Nevada School of Medicine summarizes the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystrophy in an article published this week in Science Translational Medicine.

"This is a focus article in which we summarize the impact of MG53 protein therapy as a and discuss the increasing number of new protein therapeutics being developed for the muscular dystrophies, including laminin-111 developed in our laboratory," Burkin, a pharmacological researcher and associate professor, said.

The article, "A Molecular Bandage for Diseased Muscle," co-authored by Ryan Wuebbles, a post-doctoral student in Burkin's lab in the University's Center for , is a review of the current status of therapeutic developments in the muscular dystrophy research field. These therapies represent significant strides and show great promise in treatment of muscular dystrophy.

Approaches to treat Duchenne muscular dystrophy include gene replacement therapy, gene repair and myoblast cell transfer.

In a study on MG53 by Noah Weislander and colleagues, presented in the same issue of Science Translational Medicine, it was found that mice that lack MG53 developed and exhibit defective after exercise or injury, and that MG53 facilitates rapid membrane repair to prevent damage to normal muscle.

The study suggests that treatment in combination with other protein therapies such as Burkin's laminin-111 therapy, are likely to have synergy as well for Duchenne and other muscular dystrophies.

Burkin's published research on laminin-111, a naturally occurring protein, showed it is quickly picked up in the bloodstream of mice and prevents , an important finding for Duchenne muscular dystrophy, the most common form of muscular dystrophy.

He recently co-hosted Myomatrix 2012, a conference for leading muscular dystrophy scientists and clinicians to explore and share their latest findings and data on treatment breakthroughs. The conference was held at the University of Nevada, Reno campus, where research by Burkin has led to a potential new therapy for muscular dystrophy.

Explore further: MG53 protein shown to be useful for treating traumatic tissue damage

More information: The article by Burkin and Wuebbles can be found at stm.sciencemag.org/content/4/139/139fs19.full

Burkin's work, which is funded by the National Institutes of Health, has also been featured on the website ScienCentral (www.sciencentral.com/video/2009/04/20/muscular-dystrophy-drug/). His work was published in the Proceedings of the National Academy of Sciences as well as the January 2009 edition of American Journal of Pathology.

Related Stories

MG53 protein shown to be useful for treating traumatic tissue damage

June 21, 2012
Throughout the lifecycle, injury to the body’s cells occurs naturally, as well as through trauma. Cells have the ability to repair and regenerate themselves, but a defect in the repair process can lead to cardiovascular, ...

Stem cell foundation for muscular dystrophy treatment

July 14, 2011
Research at the Australian Regenerative Medicine Institute (ARMI) at Monash University could lay the groundwork for new muscular dystrophy treatments.

Recommended for you

'Human chronobiome' study informs timing of drug delivery, precision medicine approaches

December 13, 2017
Symptoms and efficacy of medications—and indeed, many aspects of the human body itself—vary by time of day. Physicians tell patients to take their statins at bedtime because the related liver enzymes are more active during ...

Estrogen discovery could shed new light on fertility problems

December 12, 2017
Estrogen produced in the brain is necessary for ovulation in monkeys, according to researchers at the University of Wisconsin-Madison who have upended the traditional understanding of the hormonal cascade that leads to release ...

Time of day affects severity of autoimmune disease

December 12, 2017
Insights into how the body clock and time of day influence immune responses are revealed today in a study published in leading international journal Nature Communications. Understanding the effect of the interplay between ...

3-D printed microfibers could provide structure for artificially grown body parts

December 12, 2017
Much as a frame provides structural support for a house and the chassis provides strength and shape for a car, a team of Penn State engineers believe they have a way to create the structural framework for growing living tissue ...

Team identifies DNA element that may cause rare movement disorder

December 11, 2017
A team of Massachusetts General Hospital (MGH) researchers has identified a specific genetic change that may be the cause of a rare but severe neurological disorder called X-linked dystonia parkinsonism (XDP). Occurring only ...

Protein Daple coordinates single-cell and organ-wide directionality in the inner ear

December 11, 2017
Humans inherited the capacity to hear sounds thanks to structures that evolved millions of years ago. Sensory "hair cells" in the inner ear have the amazing ability to convert sound waves into electrical signals and transmit ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.