CVI Program Unit(s):
Myocyte Biology / Heart Failure
Thrombosis / Hemostasis
CVI Research Description:
Primary research focus: pathogenesis and therapy - inherited myopathies, including cardiomyopathies. In particular we have made recent progress in vector mediated gene delivery to the heart and respiratory muscles, a critical step in the develpment of durable therapy for many forms of muscular dystrophy.
In addition, my laboratory has been involved in collaborative studies (with Drs. Katherin High and Valder Arruda) on gene therapy for hemoplia, using a vascular approach to vector delivery in skeletal muscle to achieve systemic restoration of factor IX.
Toromanoff, Alice. Cherel, Yan. Guilbaud, Mickael. Penaud-Budloo, Magalie. Snyder, Richard O. Haskins, Mark E. Deschamps, Jack-Yves. Guigand, Lydie. Podevin, Guillaume. Arruda, Valder R. High, Katherine A. Stedman, Hansell H. Rolling, Fabienne. Anegon, Ignacio. Moullier, Philippe. Le Guiner, Caroline.: Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Molecular Therapy: the Journal of the American Society of Gene Therapy 16(7): 1291-9, Jul 2008.
Moore, Steven A. Shilling, Christopher J. Westra, Steven. Wall, Cheryl. Wicklund, Matthew P. Stolle, Catherine. Brown, Charlotte A. Michele, Daniel E. Piccolo, Federica. Winder, Thomas L. Stence, Aaron. Barresi, Rita. King, Nick. King, Wendy. Florence, Julaine. Campbell, Kevin P. Fenichel, Gerald M. Stedman, Hansell H. Kissel, John T. Griggs, Robert C. Pandya, Shree. Mathews, Katherine D. Pestronk, Alan. Serrano, Carmen. Darvish, Daniel. Mendell, Jerry R.: Limb-girdle muscular dystrophy in the United States. Journal of Neuropathology & Experimental Neurology 65(10): 995-1003, Oct 2006.
Bridges, Charles R. Gopal, Kapil. Holt, David E. Yarnall, Charles. Cole, Steven. Anderson, Rochelle B. Yin, Xiaoqing. Nelson, Anthony. Kozyak, Benjamin W. Wang, Zhonglin. Lesniewski, James. Su, Leonard T. Thesier, Danielle M. Sundar, Hari. Stedman, Hansell H.: Efficient myocyte gene delivery with complete cardiac surgical isolation in situ. Journal of Thoracic & Cardiovascular Surgery 130(5): 1364, Nov 2005.
Su, Leonard T. Gopal, Kapil. Wang, Zhonglin. Yin, Xiaoqing. Nelson, Anthony. Kozyak, Benjamin W. Burkman, James M. Mitchell, Marilyn A. Low, David W. Bridges, Charles R. Stedman, Hansell H.: Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector. Circulation 112(12): 1780-8, Sep 20 2005.
Arruda, Valder R. Stedman, Hansell H. Nichols, Timothy C. Haskins, Mark E. Nicholson, Matthew. Herzog, Roland W. Couto, Linda B. High, Katherine A.: Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood 105(9): 3458-64, May 1 2005.
Krupnick AS, Zhu J, Nguyen T, Kreisel1 D, Balsara1 KR, Lankford EB, Clark CC, Levine S, Stedman HH, Shrager JB. : Inspiratory loading does not accelerate dystrophy in the mdx mouse diaphragm: Implications for regenerative therapy. Journal of Applied Physics 94(2): 411-19, Feb 2003.
Kim DK, Zhu J, Kozyak BW, Burkman JM, Rubinstein NA, Lankford EB, Stedman HH, Nguyen T, Levine S, Shrager JB: Myosin heavy chain and physiological adaptation of the rat diaphragm in elastase-induced emphysema. Respiratory Research 4: 1, 2003.
Konig S, Burkman J, Fitzgerald J, Mitchell M, Su L, Stedman H. : Modular organization of phylogenetically conserved domains controlling developmental regulation of the human skeletal myosin heavy chain gene family. Journal of Biological Chemistry 277(31): 27593-27605, Aug 2 2002.
Bridges CR, Burkman JM, Malekan R, Konig SK, Chen H, Yarnell CB, Gardner TJ, Stewart AS, Stecker M, Patterson T, Stedman HH. : Global cardiac-specific transgene expression using cardiopulmonary bypass with cardiac isolation. Annals of Thoracic Surgery 73(6): 1939-46, Jun 2002.
Dejardins P, Burkman J, Shrager J, Allmond L, Stedman H. : Evolutionary implications of three novel members of the human sarcomaric myosin heavy chain gene family. Molecular Biology & Evolution 19(4): 375-393, Apr 2002.
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Last updated: 05/12/2009
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