Abstract

The purpose of the Cell Processing Core is to obtain and provide human blood cells for investigations being carried out as part of the Gene Therapy for Metabolic Disorders Program Project Grant. Cells that will be provided through this core project include peripheral blood stem cells, mononuclear cells, and umbilical cord blood cells. These cells will be obtained on a large scale from either normal donors or patients and provided in the composition obtained from the donor or processed in the laboratory to isolate sub populations such as lymphocytes or CD34++ cells. The Cell Processing Core will also provide in-vitro cell culture or clonogenic assay services for the measurement and evaluation of hematopoietic progenitor cells that have undergone gene transfer manipulations as specified in this program project. As projects move toward clinical application, the project will provide these same services for clinical trials at the GMP facility on the St. Paul Campus, the Molecular and Cellular Therapeutics Facility.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Research Program Projects (P01)
Project #
2P01HD032652-09A1
Application #
6861197
Study Section
Pediatrics Subcommittee (CHHD)
Project Start
2004-01-01
Project End
2008-12-31
Budget Start
2004-01-01
Budget End
2004-12-31
Support Year
9
Fiscal Year
2004
Total Cost
$77,605
Indirect Cost

  Institution

Name
University of Minnesota Twin Cities
Department
Type
DUNS #
555917996
City
Minneapolis
State
MN
Country
United States
Zip Code
55455

  Publications

Ou, Li; Przybilla, Michael J; Whitley, Chester B (2018) Metabolomics profiling reveals profound metabolic impairments in mice and patients with Sandhoff disease. Mol Genet Metab :
Ou, L; Przybilla, M J; Whitley, C B (2018) SAAMP 2.0: An algorithm to predict genotype-phenotype correlation of lysosomal storage diseases. Clin Genet 93:1008-1014
Aronovich, Elena L; Hyland, Kendra A; Hall, Bryan C et al. (2017) Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease. Hum Gene Ther 28:551-564
Ou, Li; Przybilla, Michael J; Whitley, Chester B (2017) Proteomic analysis of mucopolysaccharidosis I mouse brain with two-dimensional polyacrylamide gel electrophoresis. Mol Genet Metab 120:101-110
Verhaart, Ingrid E C; Robertson, Agata; Wilson, Ian J et al. (2017) Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review. Orphanet J Rare Dis 12:124
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

Showing the most recent 10 out of 92 publications