M
Mark S. Sands
Researcher at Washington University in St. Louis
Publications - 161
Citations - 9257
Mark S. Sands is an academic researcher from Washington University in St. Louis. The author has contributed to research in topics: Lysosomal storage disease & Mucopolysaccharidosis. The author has an hindex of 57, co-authored 151 publications receiving 8503 citations. Previous affiliations of Mark S. Sands include Stony Brook University & University of Washington.
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Journal ArticleDOI
AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma
Anthony Donsante,Daniel G. Miller,Yi Li,Carole Vogler,Elizabeth M. Brunt,David W. Russell,Mark S. Sands +6 more
TL;DR: It is shown that normal mice and mice with mucopolysaccharidosis VII (MPS VII) develop hepatocellular carcinoma (HCC) after neonatal injection of an AAV vector expressing b-glucuronidase, which implicate this locus in the development of HCC and raise concerns over the clinical use of AAV vectors.
Journal ArticleDOI
Gene therapy for lysosomal storage diseases.
TL;DR: The various gene replacement strategies for target organs affected in many LSDs are reviewed and the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders are described.
Journal ArticleDOI
Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
TL;DR: The data suggest that gene transfer mediated by adeno-associated virus can achieve therapeutically relevant levels of enzyme very early in life and that the rapid growth and differentiation of tissues does not limit long-term expression.
Journal ArticleDOI
Enzyme replacement therapy for murine mucopolysaccharidosis type vii
Mark S. Sands,Carole Vogler,J W Kyle,Jeffrey H. Grubb,Beth Levy,Nancy Galvin,William S. Sly,Edward H. Birkenmeier +7 more
TL;DR: Data show that recombinant beta-glucuronidase treatment begun in newborn MPS VII mice provides enzyme to most tissues and significantly reduces or prevents the accumulation of lysosomal storage during the first 6 wk of life.
Journal ArticleDOI
Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer.
John H. Wolfe,Mark S. Sands,Jane E. Barker,Babette Gwynn,Lucy B. Rowe,Carole Vogler,Edward H. Birkenmeier +6 more
TL;DR: It is shown that retroviral vector-mediated transfer of the gene to mutant stem cells results in long-term expression of low levels of β-glucuronidase which partially corrects the disease by reducing lysosomal storage in liver and spleen.