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