ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

por 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
Publicado 2021

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

por 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
Publicado 2020

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

por 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
Publicado 2018

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

por Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michael 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.
Publicado 2019