The Histopathology Core will evaluate histologic sections using standard stains and immunohistochemistry, western blots, tissue preparation and allocation for toxicology studies and biodistribution studies from all experimental studies done in mdx mice and Rhesus macaques monkeys described in Projects 1 and 2. The Histopathology Core is critical to the success of this U54 proposal for the following reasons: 1) The mission of each Project will be facilitated by the expertise of one lab with built in quality control measures to process all tissues in a consistent manner. 2) The objectivity of the results will be enhanced by separating the Pi's from the outcome measures. 3) There will be greater consistency between each of the Projects by having all tissue sections processed by the same staff. 4) Techniques available in the Histopathology Core are not common to all Pi's in each project. The Histopathology Laboratory will evaluate muscle following gene transfer from all projects to maintain consistency between projects.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Specialized Center--Cooperative Agreements (U54)
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National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
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Nationwide Children's Hospital
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Xu, Rui; Jia, Ying; Zygmunt, Deborah A et al. (2018) An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque. Mol Ther Methods Clin Dev 10:89-104
Chicoine, L G; Montgomery, C L; Bremer, W G et al. (2014) Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther 22:338-347
Chicoine, Louis G; Rodino-Klapac, Louise R; Shao, Guohong et al. (2014) Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin ?2 surrogates. Mol Ther 22:713-24
Mendell, Jerry R; Rodino-Klapac, Louise; Sahenk, Zarife et al. (2012) Gene therapy for muscular dystrophy: lessons learned and path forward. Neurosci Lett 527:90-9
Malik, Vinod; Rodino-Klapac, Louise R; Mendell, Jerry R (2012) Emerging drugs for Duchenne muscular dystrophy. Expert Opin Emerg Drugs 17:261-77
Rodino-Klapac, Louise R; Montgomery, Chrystal L; Mendell, Jerry R et al. (2011) AAV-mediated gene therapy to the isolated limb in rhesus macaques. Methods Mol Biol 709:287-98
Rodino-Klapac, Louise R; Montgomery, Chrystal L; Bremer, William G et al. (2010) Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Mol Ther 18:109-17
Chandrasekharan, Kumaran; Martin, Paul T (2010) Genetic defects in muscular dystrophy. Methods Enzymol 479:291-322
Mendell, Jerry R; Rodino-Klapac, Louise R; Rosales, Xiomara Q et al. (2010) Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol 68:629-38
Martin, Paul T; Xu, Rui; Rodino-Klapac, Louise R et al. (2009) Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. Am J Physiol Cell Physiol 296:C476-88

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