About the Authors

Alfredo D. Guerron

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Rashmi Rawat

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Arpana Sali

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Christopher F. Spurney

Affiliation Division of Cardiology, Children's National Medical Center, Washington, D. C., United States of America

Emidio Pistilli

Affiliation Pennsylvania Muscle Institute, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Hee-Jae Cha

Affiliation Department of Parasitology and Genetics, Kosin University College of Medicine, Pusan, South Korea

Gouri S. Pandey

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Ramkishore Gernapudi

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Dwight Francia

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Viken Farajian

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Diana M. Escolar

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Laura Bossi

Affiliation Transgene SA, Illkirch-Graffenstaden, France

Magali Becker

Affiliation Transgene SA, Illkirch-Graffenstaden, France

Patricia Zerr

Affiliation Transgene SA, Illkirch-Graffenstaden, France

Sabine de la Porte

Affiliation Laboratoire de Neurobiologie Cellulaire et Moléculaire, Institut Fédératif de Neurobiologie Alfred Fessard, Cedex, France

Heather Gordish-Dressman

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Terence Partridge

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Eric P. Hoffman

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Kanneboyina Nagaraju

knagaraju@cnmcresearch.org

Affiliation Center for Genetic Medicine Research, Children's National Medical Center, Department of Integrative Systems Biology, George Washington University School of Medicine and Health Sciences, Washington, D. C., United States of America

Competing Interests

This study was done by the preclinical drug testing facility as a fee-per service agreement. Laura Bossi, Megali Becker and Patricia Zerr are affiliated with Faust Pharmaceuticals. This does not alter the authors' adherence to all the PLOS one policies on sharing data and materials.

Author Contributions

Conceived and designed the experiments: DE EH KN. Performed the experiments: ADG RR AS CFS HJC GP RG DF VF EEP TP. Analyzed the data: ADG RR AS CFS LB MB PZ SdlP HGD DE EH TP KN. Contributed reagents/materials/analysis tools: AS TP. Wrote the paper: ADG HGD TP KN.