About the Authors

William E. Grose, K. Reed Clark, Danielle Griffin, Vinod Malik, Kimberly M. Shontz, Chrystal L. Montgomery, Sarah Lewis, Jerry R. Mendell, Louise R. Rodino-Klapac
Department of Pediatrics, The Ohio State University, Columbus, Ohio, United States of America

William E. Grose, K. Reed Clark, Danielle Griffin, Vinod Malik, Kimberly M. Shontz, Chrystal L. Montgomery, Sarah Lewis, Jerry R. Mendell, Louise R. Rodino-Klapac
Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, United States of America

Robert H. Brown Jr.
Department of Neurology, The University of Massachusetts Medical School, Worcester, Massachusetts, United States of America

Paul M. L. Janssen
Department of Physiology and Cell Biology, The Ohio State University, Columbus, Ohio, United States of America

Jerry R. Mendell
Department of Neurology, The Ohio State University, Columbus, Ohio, United States of America

Corresponding Authors

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

Conceived and designed the experiments: WEG JRM LRK. Performed the experiments: WEG KRC DG VM KMS CLM SL PMLJ LRK. Analyzed the data: WEG JRM LRK. Contributed reagents/materials/analysis tools: RHB. Wrote the paper: WEG JRM LRK.