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Developing Novel Therapies for Muscular Dystrophy

With a focus on limb-girdle muscular dystrophy (LGMD) and Duchenne muscular dystrophy (DMD), McColl-Lockwood Laboratory for Muscular Dystrophy Research is constantly searching for novel treatments that can make differences in the lives of patients suffering from these diseases. Each is caused by a genetic defect. There are currently no cures or effective treatments for either. Our researchers are committed to changing that.

Conducting studies that include both investigator-led and sponsor-supported, muscular dystrophy research is involved in several key programs, including:

  • Drug screening and new drug development to identify existing drugs or compounds, as well as synthesize new drugs to improve functions of dystrophic muscles and to prevent progression of muscle wasting
  • Gene therapy that includes two specific methods of delivery: viral and non-viral; therapeutic effect of gene replacement has been achieved in cell cultures and in vivo diseased models
  • Antisense therapy or exon skipping for DMD uses synthetic pieces of a gene sequence (antisense oligo) to target specific parts (exons) of the human dystrophin gene where the mutation for DMD occurs; progress is being observed in this area of research

About McColl-Lockwood Laboratory for Muscular Dystrophy Research

The McColl-Lockwood Laboratory for Muscular Dystrophy Research is a fully equipped and fully staffed facility. Our research team also collaborates with other world-renowned labs focusing on muscular dystrophy. Funding for the work being conducted by McColl-Lockwood Laboratory is received from the Muscular Dystrophy Association, NIH and other foundations. The lab was established through the Carolinas Muscular Dystrophy Research Endowment, which was created by the McColl and Lockwood families.