Brian Tseng Muscular Dystrophy Research Lab

Message from Brian Tseng

Duchenne Muscular Dystrophy (DMD) is the most common and severe progressive muscular dystrophy to affect children. Dr. Tseng's laboratory investigates the mdx mouse, an animal homolog model of DMD, which lacks the same dystrophin protein but is able to stave off the severity of muscular dystrophy.

Although there are scores of labs working to describe the function(s) of this protein, there has been a paucity of information describing how the downstream effects of dystrophin protein absence can manifest into a severe muscular dystrophy phenotype in humans but not in the mdx mice. The long-term goal will be to elucidate the differential underpinnings in the mdx mouse skeletal muscle (from lab-bench) and exploit such knowledge to translate into better understanding and therapeutics for DMD (at the bedside).

Overview: Mice without dystrophin

Dystrophin is a protein that provides strength and stability to muscles. Boys with Duchenne muscular dystrophy (DMD) do not make normal dystrophin and their muscles weaken over time. However, there are some mice (mdx) that do not have dystrophin but they do not appear crippled. Although under a microscope, some of their muscles look abnormal like boys with DMD, these mice do not lose the ability to walk. Although there are some ideas why this happens, researchers cannot explain why these mice do not lose the ability to walk. It is possible that other genes are somehow helping muscles to remain viable despite the lack of dystrophin.

Research in the Tseng lab is focused on looking for other genes in these mdx mice that helps keep their muscle function. If genes are found that keep dystrophin- deficient muscles from becoming weak, it could provide important clues for new treatments for boys with muscular dystrophy. Current techniques employed in the lab include: microarrays, bioinformatics tools, transgenic mouse technology, PCR, RNA/DNA/protein electrophoresis, immunoflourescence, confocal microscopy, magnetic resonance imaging and spectroscopy, and many others.

Philanthropy

The most challenging aspect of being both a researcher and physician is the fact that time and effort must be taken away from this important work to find funding. It is critical, yet indirectly draws away from my two primary DMD missions of medical care and research. If you know anyone or any organization looking to make tax-deductible donations, please consider designating the Tseng muscular dystrophy lab which is dedicated 100% towards muscular dystrophy research. For more information please see our Philanthropy page.

Resources

For more information about muscular dystrophy and parent support avenues, consider these websites:

Muscular Dystrophy Association (MDA) www.mdausa.org
Parent Project Muscular Dystrophy ( PPMD) http://www.parentprojectmd.org