AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction von 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

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A 3-month gene therapy single-dose IV administration pharmacology and safety study with ST-920 (isaralgagene civaparvovec) for Fabry disease in mice von Meyer, Kathleen E., Lorget, Florence, Prawdzik, Gregg, Falaleeva, Marina, Cao, Liching, Falese, Lillian, Huston, Marshall W., Hettini, Khaled, Lu, Yanmei, Ledeboer, Annemarie

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