ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Li Ou, Russell C. DeKelver, Michelle Rohde, Susan Tom, Robert Radeke, Susan J. St. Martin, Yolanda Santiago, Scott Sproul, Michelle J. Przybilla, Brenda L. Koniar, Kelly M. Podetz-Pedersen, Kanut Laoharawee, Renee D. Cooksley, Kathleen E. Meyer, Michael C. Holmes, R. Scott McIvor, Thomas Wechsler, Chester B. Whitley