The Microchemical Facility is an integral component of this program. Continuation of this work will be made possible by the use of expertise, equipment and supplies provided by this core. In addition for providing a center for intellectual interactions, experimental design, trouble-shooting and other consultation, the Microchemical Facility core operates as a service core which provides a number of biologic reagents including oligonucleotide primers heavily used in this research. In addition, the core accomplishes automated DNA sequencing of transcripts (RT-PCR) products) and cloned DNA (i.e., plasmid, retrovirus constructs and AAV vectors). The facility will also provide mutation analysis of experimental cell lines and for molecular diagnosis of patients entering into a clinical trial. The high-capacity sequencing capability will also be required to validate vector constructs and, in the Gene Scan mode, for quantitative analysis of gene insertion into target cells. The Microchemical Facility core also provides a host of other instrumentation for analysis and synthesis (e.g. for peptide sequencing, peptide synthesis, etc.) which will be used on a less frequent basis.

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Research Program Projects (P01)
Project #
5P01HD032652-12
Application #
7342408
Study Section
Pediatrics Subcommittee (CHHD)
Project Start
Project End
Budget Start
2007-01-01
Budget End
2007-12-31
Support Year
12
Fiscal Year
2007
Total Cost
$25,120
Indirect Cost
Name
University of Minnesota Twin Cities
Department
Type
DUNS #
555917996
City
Minneapolis
State
MN
Country
United States
Zip Code
55455
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
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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
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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
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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