About the Authors

Sadik H. Kassim

Affiliation Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Hui Li

Affiliation Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Luk H. Vandenberghe

Affiliation Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Christian Hinderer

Affiliation Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Peter Bell

Affiliation Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Dawn Marchadier

Affiliation Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Aisha Wilson

Affiliation Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Debra Cromley

Affiliation Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Valeska Redon

Affiliation Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Hongwei Yu

Affiliation Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

James M. Wilson

Affiliation Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Daniel J. Rader

rader@mail.med.upenn.edu

Affiliation Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America

Competing Interests

J.M.W. is a consultant to ReGenX Holdings, and is a founder of, holds equity in, and receives a grant from affiliates of ReGenX Holdings; in addition, he is an inventor on patents licensed to various biopharmaceutical companies, including affiliates of ReGenX Holdings. This does not alter the authors' adherence to all the PLoS ONE policies on sharing data and materials, as detailed online in the guide for authors.

Author Contributions

Conceived and designed the experiments: SHK HL JMW DJR. Performed the experiments: SHK CH PB AW DC VR HY. Analyzed the data: SHK LHV CH PB DM JMW DJR. Contributed reagents/materials/analysis tools: SHK HL LHV DM DJR. Wrote the paper: SHK JMW DJR. Primary Investigator: DJB. Provided extensive technical and experimental support: LHV CH PB AW DC HY. Co-Investigator: JMW. Senior Author: JMW.