The Canine Muscular Dystrophy Core (CMDC) of the proposed UNC-CH Wellstone Center will supportpilot projects on well characterized canine DMD models. Many investigators may develop only onetherapy all the way to the investigational new drug (IND) phase during their careers. These investigatorsoften will be familiar with, or have ready access to expertise for, rodent models. However, few will have abackground with analogous large animal diseases. These preliminary studies should facilitate funding forbroader hypothesis or milestone-driven research. This research could ultimately be completed through theUNC-CH National Center for Canine Models of DMD (NCDMD). The CMDC has three interrelated aimsthat should collectively support research of Wellstone Center investigators and others wishing to utilizecanine 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 fordystrophic dogs.c. Provide support to UNC-CH and/or NCDMD core facilities to include anesthesia for surgical biopsiesand imaging.
AIM 2. Work with UNC-CH and/or NCDMD core facilities jo develop and refine biomarkers that can be usedto determine efficacy of proposed DMD treatments.:a. Facilitate performance of morphologic, immunohistochemical, ultrastructurai and biodistributionstudies needed by investigators.b. Facilitate development of noninvasive functional tests that can be used serially over time to define thenatural history and response to treatment of canine DMD models.c. Facilitate establishment of user-defined magnetic resonance imaging (MRI) protocols by providingimaging services and image analysis tools.
AIM 3. Collaborate with Wellstone Center investigators and others to conduct pilot projects that bolstertranslational research focused on therapeutic strategies for DMD.
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