Search Results - "Hartung, S D"

Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase gene by Hartung, S D, Reddy, R G, Whitley, C B, McIvor, R S

“…Mucopolysaccharidosis type I (MPS I), a deficiency in the lysosomal enzyme alpha-L-iduronidase (IDUA), is characterized by skeletal abnormalities,…”
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In Vivo Transduction of Cerebellar Purkinje Cells Using Adeno-Associated Virus Vectors by Kaemmerer, William F., Reddy, Rukmini G., Warlick, Christopher A., Hartung, Seth D., McIvor, R. Scott, Low, Walter C.

“…We investigated whether adenovirus or adeno-associated virus vectors can transduce cerebellar Purkinje cells (PCs) in vivo. Mice were injected in the deep…”
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Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene by Hartung, Seth D, Frandsen, Joel L, Pan, Dao, Koniar, Brenda L, Graupman, Patrick, Gunther, Roland, Low, Walter C, Whitley, Chester B, McIvor, R Scott

“…Murine models of lysosomal storage diseases provide an opportunity to evaluate the potential for gene therapy to prevent systemic manifestations of the…”
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