Mark Haskins, VMD, PhD
Professor, Pathobiology

Gene Therapy and Vaccines Program

Address

310A Rosenthal Building/Vet
3800 Spruce Street
Philadelphia, PA 19104-6051

Office tel.: 215 898-4852
Fax: 215 898-0719
E-mail: mhaskins@vet.upenn.edu

Pennsylvania State University: BS (Pre-Vet), 1966.

University of Pennsylvania: VMD (Veterinary Medicine), 1969.

Drexel University: MS (Biomedical Engineering), 1974.

University of Pennsylvania: PhD (Pathology), 1979.

Research Interests

• Discovery, characterization, and therapy of large animal models of human genetic disease.

Key words: Dog, cat, lysosomal storage disease.

Description of Research

In collaboration with others, a series of metabolic diseases caused by deficient enzyme activities are being studied, including mucopolysaccharidoses (MPS) I, VI, and VII (alpha-L-iduronidase, 4-sulfatase, and ß-glucuronidase deficiencies, respectively), alpha mannosidosis (alpha-mannosidase deficiency), I-cell disease (N-acetylglucosamine-1-phosphotransferase), Krabbe disease (galactosylceramidase), and acute intermittent porphyria (PBG-deaminase deficiency). Five of these diseases are lysosomal storage disorders with clinical phenotypes in the animals that are the same as in affected children. Experiments include administering recombinant human alpha-L-iduronidase intravenously to cats with MPS I and gene therapy using retroviral and AAV vectors. The gene therapy experiments are being performed in MPS I cats and dogs, MPS VI cats, and MPS VII dogs using the species-specific cDNAs except feline MPS I. The somatic cell gene therapy experiments include a) intraocular injections of AAV-4-sulfatase vector virus in MPS VI cats, b) neonatal intravenous retrovirus gene therapy in MPS I and VI cats, MPS I dogs, and MPS VII dogs, and c) intramuscular AAV vectors containing the feline 4S cDNA and human SUMF-1 cDNA.

Selected Publications

Simonaro, C., D’Angelo, M., He, X., Eliyahu, E., Shtraizent, N., Haskins, M.E., and Schuchman, E.H. (2008) Mechanisms of glycosaminoglycan-mediated disease: Implications for the mucopolysaccharidoses and other connective tissue diseases. Am J Pathol 172:112-122.

Tessitore, A, Faella, A., O’Malley, T. Doria, M. Cotugno, G. Kunieda, T., Mararese, G., Haskins, M., Auricchio, A. (2008) Liver but not muscle transduced with AAV results in secretion of therapeutic levels of ARSB and amelioration of the MPS VI phenotype in animal models. Mol Ther 16:30-37.

Sleeper, M.M., Kusiak, C.M., O’Donnell, P., Bryan C., Ponder, K.P., Haskins, M.E. (2008) Clinical characterization of cardiovascular abnormalities associated with feline mucopolysaccharidosis I and VI. J Inherit Metab Dis. in press

Vite, C.H., Ding, W., Bryan, C., O’Donnell, P., Cullen, K., Aleman, D., Cozzi, F., Haskins, M.E., van Winkle, T (2008) Progression of neurologic and hepatic abnormalities in feline Niemann- Pick type C disease Ped Res, in press

Herati, R.S., Knox, V.W., Ma, X., O’Donnell, P., D’Angelo, M., Haskins, M. (2008) Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy. Molec Genet Metab, in press.

Search PubMed for more articles

Lab

Rotation Projects

Please see Dr. Haskins.

Lab personnel

Thomas O'Malley, Lab Research Specialist
Ping Wang, Lab Reseach Specialist
Ulana Prociuk, Lab Research Specialist
Angie Huff , Lab Reseach Specialist
Patty O'Donnell, Animal Research Specialist