A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Courtney S. Young, Michael R. Hicks, Natalia V. Ermolova, Haruko Nakano, Majib Jan, Shahab Younesi, Saravanan Karumbayaram, Chino Kumagai-Creese, Derek Wang, Jerome A. Zack, Donald B. Kohn, Atsushi Nakano, Stanley F. Nelson, M. Carrie Miceli, Melissa J. Spencer & April D. Pyle

Thursday, February 11, 2016
Published in Cell Stem Cell