The Canine Muscular Dystrophy Core (CMDC) of the proposed UNC-CH Wellstone Center will support pilot projects on well characterized canine DMD models. Many investigators may develop only one therapy all the way to the investigational new drug (IND) phase during their careers. These investigators often will be familiar with, or have ready access to expertise for, rodent models. However, few will have a background with analogous large animal diseases. These preliminary studies should facilitate funding for broader hypothesis or milestone-driven research. This research could ultimately be completed through the UNC-CH National Center for Canine Models of DMD (NCDMD). The CMDC has three interrelated aims that should collectively support research of Wellstone Center investigators and others wishing to utilize canine DMD models in preclinical research.
AIM 1. Perpetuate canine models of DMD to meet investigator demands for this important preclinical model. a. Monitor estrus and breed carriers to produce dogs for preclinical studies. b. Work with the UNC-CH Department of Laboratory Animal Medicine to provide specialized care for dystrophic dogs. c. Provide support to UNC-CH and/or NCDMD core facilities to include anesthesia for surgical biopsies and imaging.
AIM 2. Work with UNC-CH and/or NCDMD core facilities jo develop and refine biomarkers that can be used to determine efficacy of proposed DMD treatments.: a. Facilitate performance of morphologic, immunohistochemical, ultrastructurai and biodistribution studies needed by investigators. b. Facilitate development of noninvasive functional tests that can be used serially over time to define the natural history and response to treatment of canine DMD models. c. Facilitate establishment of user-defined magnetic resonance imaging (MRI) protocols by providing imaging services and image analysis tools.
AIM 3. Collaborate with Wellstone Center investigators and others to conduct pilot projects that bolster translational research focused on therapeutic strategies for DMD.
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