The objective of the Research Animal Care and Medicine Core is to provide the appropriate animal models and technical and pathology support to the research projects of this grant in a timely and cost-effective manner. The multiple specialized animal models of genetic dysfunction used for each of these projects require expert colony management including animal and veterinary care, breeding management, housing, veterinary preventive medicine and diagnostic support, as well as technical expertise in experimental manipulation of all ages of mice and dogs. Analysis of housing needs, procurement and breeding options, and intensive management and monitoring of in-house mouse production colonies are necessary forthe smooth functioning ofthe specific aims ofthe research projects. The Research Animal Care and Medicine Core will provide veterinary oversight and individualized care and programming for the animal models integral to each project. This Core will interact closely with the other Cores to provide the most appropriate samples for prompt genotyping and individual animal identification. Animal Core staff will also provide technical support (e.g. intravascular administration of rDNA to newborn mice, post-procedural care for mice and dogs, clinical, hematologic and clinical pathologic evaluation of animals on project experiments, therapeutic intervention if necessary, harvesting of brain tissues for evaluation by Core B) and routine research pathology support. Quality control is focused on ensuring the genetic integrity of mice by genotyping and record-keeping and on preventing confounding effects from intercurrent infectious and noninfectious disease. The latter is accomplished by specific pathogen-free housing, by an intensive serology surveillance program to detect the presence of potential pathogens, close monitoring ofthe animals' health, and diagnostic pathology as needed. Prevention of confounding effects of infectious and non- infectious conditions in dogs will be ensured by proper choice of sources of dogs and intensive health monitoring and treatment when appropriate.

Public Health Relevance

Provision of genotypically and phenotypically appropriate animal models and prevention of confounding effects of intercurrent health problems in animal models is crucial to the success ofthe projects in advancing the understanding and treatment of MPS diseases in people.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Research Program Projects (P01)
Project #
Application #
Study Section
Special Emphasis Panel (ZHD1-DSR-Z)
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
University of Minnesota Twin Cities
United States
Zip Code
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
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
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; 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