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

Kanut Laoharawee, Russell C. DeKelver, Kelly M. Podetz-Pedersen, Michelle Rohde, Scott Sproul, Hoang Oanh Nguyen, Tam Nguyen, Susan J. St. Martin, Li Ou, Susan Tom, Robert Radeke, Kathleen E. Meyer, Michael C. Holmes, Chester B. Whitley, Thomas Wechsler, R. Scott McIvor