The Administrative Core (Core A) exists to facilitate and coordinate the components of the Program toward fulfilling the Program's mission;the overall objective of the Program is the development of innovative methods of gene therapy for metabolic disorders, and especially the mucopolysaccharidosis (MPS) diseases. The Program is comprised of three research projects and three supportive cores. Core A will provide centralized administrative functions for the Program. To that end, the Administrative Core will implement efficient use of the resources, encourage synergism, organize meetings of Program participants and of the External Advisory Committee. The core will also facilitate maintain and update databases that support Program activities and serve as a central communications hub for the investigators, research staff, and support personnel, as well as provide centralized financial and accounting services support.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Research Program Projects (P01)
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Special Emphasis Panel (ZHD1-DSR-Z)
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University of Minnesota Twin Cities
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Aronovich, Elena L; Hackett, Perry B (2015) Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier. Mol Genet Metab 114:83-93
Ou, Li; Herzog, Tyler L; Wilmot, Carrie M et al. (2014) Standardization of *-L-iduronidase enzyme assay with Michaelis-Menten kinetics. Mol Genet Metab 111:113-5
Carpentier, Claire E; Schreifels, Jeffrey M; Aronovich, Elena L et al. (2014) NMR structural analysis of Sleeping Beauty transposase binding to DNA. Protein Sci 23:23-33
Ou, Li; Herzog, Tyler; Koniar, Brenda L et al. (2014) High-dose enzyme replacement therapy in murine Hurler syndrome. Mol Genet Metab 111:116-22
Janson, Christopher G; Romanova, Liudmila G; Leone, Paola et al. (2014) Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-?-L-Iduronidase for Hurler Disease. Neurosurgery 74:99-111
Zhao, Gengxiang; Allewell, Norma M; Tuchman, Mendel et al. (2013) Structure of the complex of Neisseria gonorrhoeae N-acetyl-L-glutamate synthase with a bound bisubstrate analog. Biochem Biophys Res Commun 430:1253-8
Hackett, Perry B; Largaespada, David A; Switzer, Kirsten C et al. (2013) Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy. Transl Res 161:265-83
Wolf, Daniel A; Hanson, Leah R; Aronovich, Elena L et al. (2012) Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration. Mol Genet Metab 106:131-4
Wolf, Daniel A; Lenander, Andrew W; Nan, Zhenhong et al. (2011) Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I. Neurobiol Dis 43:123-33
Shi, Dashuang; Li, Yongdong; Cabrera-Luque, Juan et al. (2011) A novel N-acetylglutamate synthase architecture revealed by the crystal structure of the bifunctional enzyme from Maricaulis maris. PLoS One 6:e28825

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