McColl-Lockwood Laboratory for Muscular Dystrophy Research
The McColl-Lockwood laboratories were established in 2005 with a generous and ongoing research endowment grant from the Carolinas Muscular Dystrophy Research Endowment, which was created by the McColl and Lockwood families as well as the Carolinas HealthCare Foundation. The Laboratory is part of the Carolinas Neuromuscular/ALS Center associated with the Department of Neurology at Carolinas Medical Center, which provides service to patients in the region and beyond.
The Department has established and maintained an active large clinical database and the essential infrastructure for clinical trial. The McColl-Lockwood Laboratory focuses on the translational research of muscular dystrophy, specifically limb-girdle muscular dystrophy (LGMD) and Duchenne muscular dystrophy (DMD). These diseases are caused by genetic defects (gene mutations) that disrupt normal muscle functions, resulting in weakening of muscle strength. Currently, there is no cure or effective treatment for LGMD and DMD.
The goal of the Laboratory is to develop novel therapeutic approaches for the treatment of the diseases, with emphasis on pre-clinical testing, new drug development, gene therapy and other molecular therapies. To facilitate the process, multiple collaborations have been established among the Laboratory and other internationally renowned researchers. The Laboratory has made significant progress in the following areas:
- Several in vitro and in vivo models have been created for the study of fkrp gene and LGMD2I.
- Programs for drug screening and design are well underway for identification of promising candidate pharmaceutical agents for the diseases.
- Effective non-viral gene/oligonucleotide delivery systems, vital for successful application of gene therapy, are being developed.
- Research of antisense oligonucleotide therapy for DMD has been at the fore front with the aim to establish long-term efficacy of the therapy, identify highly effective antisense drug targeting individual dystrophin exon and develop AOs into therapeutic drugs.
- Unique cell cultures and animal models have been established for test and validation of new therapies for both dystroglycanopathy and DMD.
In addition to substantial annual support from the Carolinas Muscular Dystrophy Research Endowment, the Laboratory receives funding from the Muscular Dystrophy Association, US Department of Defense, North Carolina Biotechnology Center, and NIH.