About the Authors

Dilay Hazal Ayhan

Contributed equally to this work with: Dilay Hazal Ayhan, Yusuf Talha Tamer

Affiliations Green Center for Systems Biology, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America, Department of Biochemistry and Molecular Biology, University of Massachusetts Amherst, Amherst, Massachusetts, United States of America

Yusuf Talha Tamer

Contributed equally to this work with: Dilay Hazal Ayhan, Yusuf Talha Tamer

Affiliations Green Center for Systems Biology, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America, Department of Molecular Biophysics, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America

Mohammed Akbar

Affiliation Green Center for Systems Biology, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America

Stacey M. Bailey

Affiliation Sarepta Therapeutics, Cambridge, Massachusetts, United States of America

Michael Wong

Affiliations Sarepta Therapeutics, Cambridge, Massachusetts, United States of America, Harvard Medical School, Cambridge, Massachusetts, United States of America

Seth M. Daly

Affiliation Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America

David E. Greenberg

David.Greenberg@UTSouthwestern.edu (DEG); Erdal.Toprak@UTSouthwestern.edu (ET)

Affiliations Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America, Department of Microbiology, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America

Erdal Toprak

David.Greenberg@UTSouthwestern.edu (DEG); Erdal.Toprak@UTSouthwestern.edu (ET)

Affiliations Green Center for Systems Biology, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America, Department of Pharmacology, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America

Competing Interests

MW and SMB are employees of Sarepta Therapeutics that hold numerous patents on the methods of synthesis and use of PPMOs. DEG receives research support from Sarepta Therapeutics, holds several patents related to PPMOs, and receives license-related royalties for these. All other authors declare no competing financial interest.

Author Contributions

Conceptualization: DHA YTT DEG ET. Data curation: DHA YTT ET. Formal analysis: DHA YTT MA SMD DEG ET. Funding acquisition: DEG ET. Investigation: DHA YTT MA SMB SMD DEG ET. Methodology: DHA YTT MA SMB SMD DEG ET. Project administration: DEG ET. Software: DHA YTT. Supervision: DEG ET. Validation: DHA YTT MA SMD DEG ET. Visualization: DHA YTT ET. Writing – original draft: DHA YTT SMD DEG ET. Writing – review & editing: MW.