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.
|Chai, Zheng; Samulski, R Jude; Li, Chengwen (2018) Nab Escaping AAV Mutants Isolated from Mouse Muscles. Bio Protoc 8:|
|Li, Chengwen; Wu, Shuqing; Albright, Blake et al. (2016) Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer. Mol Ther 24:53-65|
|Fan, Zheng; Kocis, Keith; Valley, Robert et al. (2015) High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline. Hum Gene Ther 26:614-21|
|Fan, Zheng; Wang, Jiahui; Ahn, Mihye et al. (2014) Characteristics of magnetic resonance imaging biomarkers in a natural history study of golden retriever muscular dystrophy. Neuromuscul Disord 24:178-91|
|Qiao, Chunping; Li, Chengwen; Zhao, Chunxia et al. (2014) K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo. Hum Gene Ther Methods 25:33-9|
|Powers, William J (2014) Intravenous thrombolysis of basilar artery thrombosis. Ann Neurol 75:456-7|
|Mitchell, Angela M; Hirsch, Matthew L; Li, Chengwen et al. (2014) Promyelocytic leukemia protein is a cell-intrinsic factor inhibiting parvovirus DNA replication. J Virol 88:925-36|
|Asokan, Aravind; Samulski, R Jude (2013) An emerging adeno-associated viral vector pipeline for cardiac gene therapy. Hum Gene Ther 24:906-13|
|Gray, S J; Nagabhushan Kalburgi, S; McCown, T J et al. (2013) Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther 20:450-9|
|He, Yi; Weinberg, Marc S; Hirsch, Matt et al. (2013) Kinetics of adeno-associated virus serotype 2 (AAV2) and AAV8 capsid antigen presentation in vivo are identical. Hum Gene Ther 24:545-53|
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