Abstract

The overall goal of the MDCRC Training and Education Core is to provide trainees with the skills necessary to translate advances in basic science muscular disease research into well-designed human clinical trials. To be successful the trainees must have an understanding of the concepts, features, and limitations of the interventions to be used in clinical studies, the biology of the inherited disease target to be approached, the ethics of human and animal research and the importance of well designed clinical trials to answer specific questions. These concepts will be taught through a series of formal courses and seminars, discussions with scientists and ethicists, regular interactions with clinical scientists and interactions with basic scientists. Mentored research under the direction of the MDCRC director and co-director will present trainees with the practical skills needed to pursue careers in translational research.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
5U54AR056953-05
Application #
8380618
Study Section
Special Emphasis Panel (ZAR1-KM-J)
Project Start
Project End
2014-02-28
Budget Start
2012-09-01
Budget End
2013-08-31
Support Year
5
Fiscal Year
2012
Total Cost
$173,739
Indirect Cost
$56,548

  Institution

Name
University of North Carolina Chapel Hill
Department
Type
DUNS #
608195277
City
Chapel Hill
State
NC
Country
United States
Zip Code
27599

  Publications

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
Hirsch, Matthew L; Li, Chengwen; Bellon, Isabella et al. (2013) Oversized AAV transductifon is mediated via a DNA-PKcs-independent, Rad51C-dependent repair pathway. Mol Ther 21:2205-16
Mitchell, Angela M; Li, Chengwen; Samulski, R Jude (2013) Arsenic trioxide stabilizes accumulations of adeno-associated virus virions at the perinuclear region, increasing transduction in vitro and in vivo. J Virol 87:4571-83
Ferreira, J R; Hirsch, M L; Zhang, L et al. (2013) Three-dimensional multipotent progenitor cell aggregates for expansion, osteogenic differentiation and 'in vivo' tracing with AAV vector serotype 6. Gene Ther 20:158-68

Showing the most recent 10 out of 38 publications