AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
Yasuda, Makiko, Huston, Marshall W., Pagant, Silvere, Gan, Lin, St. Martin, Susan, Sproul, Scott, Richards, Daniel, Ballaron, Stephen, Hettini, Khaled, Ledeboer, Annemarie, Falese, Lillian, Cao, Liching, Lu, Yanmei, Holmes, Michael C., Meyer, Kathleen, Desnick, Robert J., Wechsler, Thomas
Published in Molecular therapy. Methods & clinical development (11.09.2020)
Published in Molecular therapy. Methods & clinical development (11.09.2020)
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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michelle J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, Whitley, Chester B.
Published in Molecular therapy (02.01.2019)
Published in Molecular therapy (02.01.2019)
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Chemical Induction of Misfolded Prion Protein Conformers in Cell Culture
Ghaemmaghami, Sina, Ullman, Julie, Ahn, Misol, St. Martin, Susan, Prusiner, Stanley B.
Published in The Journal of biological chemistry (02.04.2010)
Published in The Journal of biological chemistry (02.04.2010)
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A Survey of Antiprion Compounds Reveals the Prevalence of Non-PrP Molecular Targets
Poncet-Montange, Guillaume, St. Martin, Susan J., Bogatova, Olga V., Prusiner, Stanley B., Shoichet, Brian K., Ghaemmaghami, Sina
Published in The Journal of biological chemistry (05.08.2011)
Published in The Journal of biological chemistry (05.08.2011)
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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
Laoharawee, Kanut, DeKelver, Russell C., Podetz-Pedersen, Kelly M., Rohde, Michelle, Sproul, Scott, Nguyen, Hoang-Oanh, Nguyen, Tam, St. Martin, Susan J., Ou, Li, Tom, Susan, Radeke, Robert, Meyer, Kathleen E., Holmes, Michael C., Whitley, Chester B., Wechsler, Thomas, McIvor, R. Scott
Published in Molecular therapy (04.04.2018)
Published in Molecular therapy (04.04.2018)
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ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice
Pagant, Silvere, Huston, Marshall W., Moreira, Luciana, Gan, Lin, St Martin, Susan, Sproul, Scott, Holmes, Michael C., Meyer, Kathleen, Wechsler, Thomas, Desnick, Robert J., Yasuda, Makiko
Published in Molecular therapy (03.11.2021)
Published in Molecular therapy (03.11.2021)
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Liver-targeted AAV gene therapy vectors produced by a clinical scale manufacturing process result in high, continuous therapeutic levels of enzyme activity and effective substrate reduction in mouse model of Fabry disease
Huston, Marshall W., Yasuda, Makiko, Pagant, Silvere, Martin, Susan St, Cao, Liching, Falese, Lillian, Meyer, Kathleen, Desnick, Robert J., Wechsler, Thomas
Published in Molecular genetics and metabolism (01.02.2019)
Published in Molecular genetics and metabolism (01.02.2019)
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Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction
Huston, Marshall W., Yasuda, Makiko, Pagant, Silvere, Martin, Susan St, Sproul, Scott, DeKelver, Russell, Holmes, Michael C., Desnick, Robert J., Wechsler, Thomas
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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ZFN-mediated in vivo genome editing results in therapeutic levels of α-galactosidase A and effective substrate reduction in Fabry knockout mice
Pagant, Silvere, Huston, Marshall W., Yasuda, Makiko, Martin, Susan St, Sproul, Scott, DeKelver, Russell, Holmes, Michael C., Wechsler, Thomas, Desnick, Robert J.
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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Liver-based expression of the human alpha-galactosidase A gene (GLA) in a murine Fabry model results in continuous supra-physiological enzyme activity and effective substrate reduction
Huston, Marshall W., Yasuda, Makiko, Pagant, Silvere, St. Martin, Susan, Sproul, Scott, Santiago, Yolanda, DeKelver, Russel, Holmes, Michael C., Desnick, Robert, Wechsler, Thomas
Published in Molecular genetics and metabolism (01.01.2017)
Published in Molecular genetics and metabolism (01.01.2017)
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1333 MICROBIAL PROFILE OF CHRONICALLY INTUBATED NEWBORN INFANTS
Bartoletti, Albert, Lepow, Martha, Martin, Susan St, Venezia, Richard, Hipp, Sally
Published in Pediatric research (01.04.1985)
Published in Pediatric research (01.04.1985)
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1332 UREAPLASMA UREALYTICUM AND NEWBORN RESPIRATORY DISTRESS
Bartoletti, Albert, Lepow, Martha, Martin, Susan St, Venezia, Richard, Hipp, Sally
Published in Pediatric research (01.04.1985)
Published in Pediatric research (01.04.1985)
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Microenvironmental Immunoregulation: Possible Role of Contrasuppressor Cells in Maintaining Immune Responses in Gut-Associated Lymphoid Tissues
Green, Douglas R., Gold, Jerome, St. Martin, Susan, Gershon, Robyn, Gershon, Richard K.
Published in Proceedings of the National Academy of Sciences - PNAS (01.02.1982)
Published in Proceedings of the National Academy of Sciences - PNAS (01.02.1982)
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