CMT is a genetic neurological disorder that causes damage to the peripheral nerves, the bundles of nerve cell fibers that connect the brain and spinal cord to muscles and sensory organs. There are several different types of the disease; Burgess studies CMT-2D, which is characterized by muscle weakness and loss or changes in sensation in extremities.
CMT is associated with a mutation in a gene known as GARS. Burgess and co-investigator Scott Harper, associate professor at Nationwide Children’s Hospital Center for Gene Therapy in Columbus, Ohio, will test new gene therapy approach to specifically block the altered form of the GARS gene in a newly developed mouse model for CMT-2D.
“It is really important that the MDA is willing to support this kind of high-risk, high-return research on rare and orphan diseases such as Charcot-Marie-Tooth disease,” Burgess comments. “This project has the potential to develop personalized gene therapy strategies for treating otherwise intractable diseases.”
The award represents a portion of $10 million in new research grants that the MDA has awarded in new research grants to researchers studying muscular dystrophy, ALS and related muscle-debilitating diseases. Through its investigator-initiated application process, MDA received 350 grant requests, the most ever in its 65-year history of funding basic, translational and clinical research. It awarded grants to 36 researchers, including Burgess.
“These new grants are potential game-changers, a signal of our passionate resolve to helping kids and adults fighting neuromuscular diseases live longer and grow stronger,” says Valerie Cwik, M.D., MDA's executive vice president and chief medical & scientific officer. “There’s more new drug discovery underway than ever before, and we’re determined to double the number of promising human clinical trials in the next five years."
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.
The Jackson Laboratory is an independent, nonprofit biomedical research institution and National Cancer Institute-designated Cancer Center based in Bar Harbor, Maine, with a facility in Sacramento, Calif., and The Jackson Laboratory for Genomic Medicine in Farmington, Conn. It employs 1,700 staff, and its mission is to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health.