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.
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
Karyn Cullen, Animal Technician
PRIMARY AREAS OF RESEARCH COMPETENCE
Pathology
Genetics
Ellinwood, N.M., Vite, C.H., and Haskins, M.E. Gene therapy for lysosomal storage diseases: The lessons and promise of animal models J. Gene Medicine 6: 481-506, 2004.Mango, RL, Xu L, Sands, MS, Vogler, C, Seiler, G, Schwarz, T, Haskins, ME, Ponder, KP Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs Mol Genet Metab 82: 4-19, 2004.Sleeper, M.M., Fornasari, B., Ellinwood, N.M., Weil, M.A., Melniczek, J., O’Malley, T.M., Sammaroc, C.D., Xu, L., Ponder, K.P., and Haskins, M.E. Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII Circulation : , 2004.Suter, S.E., Gouthro, T.A., McSweeney, P.A., Nash, R.A., Haskins, M.E., Felsburg, P.J., Henthorn, P.S. Isolation and characterization of pediatric canine bone marrow CD34+ cells. Vet. Immunol. and Immunopathology : , 2004.Xu, L., O’Malley, T., Sands, M.S., Wang, B., Meyerrose, T., Haskins, M., Ponder, K.P. In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice Molecular Therapy : , 2004.
BA (Pre-Vet) Pennsylvania State University, 1966VMD (Veterinary Medicine) University of Pennsylvania, 1969MS (Biomedical Engineering) Drexel University, 1974Ph.D (Pathology) University of Pennsylvania, 1979
Drexel University, Philadelphia, PA.
(1971)
1971 National Heart and Lung Institute Research Fellow,