The overarching goal of the National Center for Canine Models of Duchenne muscular dystrophy (NCDMD) at the University of North Carolina-Chapel Hill is to develop and sustain dog models of DMD and to provide high quality facilities and services in compliance with GLP standards to support pre-investigational new drug (IND) applications and, thus, meet the increasing demand to utilize golden retriever muscular dystrophy (GRMD) and other canine models of DMD in preclinical studies required for human clinical trials.  The essential services provided by the NCDMD will reduce costs of assessing efficacy, ensure that results are consistent among investigators, and facilitate rapid translation to clinical care.  In addition, because the breeding colonies and core infrastructure of the NCDMD is supported through a NINDS/NIAMS Co-operative Program in Translational Research (U24) grant, fees charged to investigators are reduced.  The U24 grant has two major Aims:

Aim 1. ESTABLISH INFRASTRUCTURE AND ORGANIZATIONAL COMPONENTS OF THE NCDMD, INCLUDING:

1. Administrative and Quality Coordination Office (AQCO), which conducts and manages day-to-day aspects of the primary CMDF (below) and corresponding service facilities and oversees each facility’s quality control program.
2. A Steering Committee, which oversees issues pertaining to program selection, program management, program milestones and advancement, intellectual property, public dissemination of data as well as refining and adapting NCDMD strategies in response to possible changing definitions of “best practices.”
3. An Operations Committee, which manages day-to-day activities of the NCDMD. This committee works in tandem with the Steering Committee to set policies of the NCDMD and its cores, to include development of needed procedures and fees to be charged. Most importantly, the Operations Committee meets monthly to review progress of ongoing projects conducted through the NCDMD.
4. A Canine Muscular Dystrophy Facility (CMDF), which produces and maintains a breeding colony of well-characterized dogs with genetically-determined muscular dystrophy.
5. Service facilities including the Physiology Testing Facility (PTF), Histology & Molecular Services Facility (H&MSF) and Large Animal Imaging Facility (LAIF), which provide specialized support services for research projects using the dogs bred and maintained at the CMDF.

Aim 2. CONDUCT TRANSLATIONAL RESEARCH FOCUSED ON THERAPEUTIC STRATEGIES FOR DMD.

This research will lead to identification of promising candidates for advancement to IND/clinical trials. Projects to be conducted through the NCDMD will be chosen by the NCDMD Steering Committee based on stringent quantifiable criteria. Continued support of these projects will be contingent on completion of milestones and validation of the need for extending services offered by the NCDMD.