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