Muscle Development and Regeneration Program

Heart and skeletal muscle disorders contribute to substantial mortality and morbidity not only for adults but also for children and young adults.  The new Muscle Development and Regeneration Program (MDR) will focus on understanding the etiology and developing drug and cell-based therapies for these diseases with emphasis on 3 areas of research:  1) the molecular genetic basis of congenital heart disease (CHD), 2) development of strategies for regeneration of myocardial and skeletal muscle cells for treatment of degenerative diseases, and 3) developmental basis and pathogenesis of myopathies, including muscle cell structural anomalies and pathological myocardial hypertrophy. The program will build on current strengths in mouse transgenic and surgical models of heart disease, including CHDs, research on muscle stem and progenitor cells, and discovery of small molecule and microRNA regulators of muscle development that will be used to develop probes of muscle disease and drugs for therapy.  The program’s primary goal of understanding muscle biology and disease at a developmental, genetic and cell signaling level complements but is distinct from the main interests of other BIMR programs.  In particular, the MDR focus complements the interests of the Heart Pathophysiology program (Lake Nona), which emphasizes adult heart disease, the Development and Aging program (La Jolla), which focuses on basic developmental biology and aging mechanisms, and the Stem Cell and Regeneration program (La Jolla), which focuses on broad issues of stem cell potency, differentiation and applications in regenerative medicine. The MDR program expects to foster interactions and coordinate recruitment with these programs as well as with the RNA Biology program in the Sanford Center. In addition, the program will seek to strengthen ties to partners in Florida and Sioux Falls for sourcing clinical material, and to investigators in the Cardiovascular Center and Bioengineering Department at UCSD to develop multidisciplinary approaches to the treatment of cardiac and skeletal muscle disorders.