Core B: Neuro-Histology and Behavior Core. The overall objective of the Program Project is to study the effects of various gene constructs for treating lysosomal metabolic disorders in mice. If the gene constructs are capable of correcting the metabolic disorders then the treated mice must be studied to determine their phenotype. This will be done histologically to determine if cells express the appropriate enzymes, and whether the pathology has been corrected. Animal phenotype with respect to neurological/behavioral function and auditory neurophysiology will also be evaluated by this core. These services will aid in addressing the central hypothesis of the program project, i.e., that gene therapy can correct neurological deficits associated with lysosomal storage disorders. Also, mice that are homozygous for these disorders must be genotyped for proper identification. Core B will provide this genotyping service.

Public Health Relevance

The functions of the Neuro-Histology and Behavior Core are integral to the research performed by the Program. This Core provides support for all aspects of animal behavioral assessment, neurohistology, and genotyping for Projects I, II, and III.

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
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

Showing the most recent 10 out of 72 publications