The purpose of the Viral Vector Core is to provide packaged virus vectors for investigators being carried out as a part of the Gene Therapy for Metabolic Disorders program. Recombinant DNA manipulations will be carried out in the laboratories of the individual investigators to generate specific vector constructs. These vector constructs will then be packaged in the Viral Vector Core, relying on extensive experience of the vector core laboratory in the procedures carried out for these preparations. Vector types to be prepared for this program include recombinant adeno- associated virus vectors, lentivirus vectors, and adenovirus vectors for Projects 2, 3, and 4, respectively. The Viral Vector Core will also provide guidance on vector construct design and analysis for vector-mediated gene transfer into target cell populations. As each project moves toward a clinical trial (e.g., Project 3), work of the Vector Core may include GLP testing of materials (e.g., packaging cell lines) being sent to the University's GMP gene therapy manufacturing facility, the Gene Therapeutics unit (Dr. Schott McIvor, Director) at the Molecular and Cellular Therapeutics Facility. In this case, the facilities and staff of this large facility may assume responsibility (and funding) for the same vector production activities at the alternate location on the St. Paul Campus.

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Research Program Projects (P01)
Project #
5P01HD032652-07
Application #
6442595
Study Section
Pediatrics Subcommittee (CHHD)
Project Start
2001-01-01
Project End
2001-12-31
Budget Start
Budget End
Support Year
7
Fiscal Year
2001
Total Cost
$106,204
Indirect Cost
Name
University of Minnesota Twin Cities
Department
Type
DUNS #
168559177
City
Minneapolis
State
MN
Country
United States
Zip Code
55455
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Ou, Li; Przybilla, Michael J; Whitley, Chester B (2017) Phenotype prediction for mucopolysaccharidosis type I by in silico analysis. Orphanet J Rare Dis 12:125
Hyland, Kendra A; Aronovich, Elena L; Olson, Erik R et al. (2017) Transgene Expression in Dogs After Liver-Directed Hydrodynamic Delivery of Sleeping Beauty Transposons Using Balloon Catheters. Hum Gene Ther 28:541-550
Ou, Li; Przybilla, Michael J; Koniar, Brenda L et al. (2016) Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I. Mol Genet Metab Rep 8:87-93
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
Satzer, David; DiBartolomeo, Christina; Ritchie, Michael M et al. (2015) Assessment of dysmyelination with RAFFn MRI: application to murine MPS I. PLoS One 10:e0116788

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