About the Authors

Soumitra Roy

Current address: Crucell Holland, Leiden, The Netherlands

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

Roberto Calcedo

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

Angelica Medina-Jaszek

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

Martin Keough

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

Hui Peng

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

James M. Wilson

wilsonjm@mail.med.upenn.edu

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

Competing Interests

SR is an inventor on patents licensed to various biopharmaceutical companies. JMW 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 work was also partially funded by a commercial source (GlaxoSmithKline). 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: SR RC MK JMW. Performed the experiments: RC AM-J MK HP. Analyzed the data: SR MK JMW. Wrote the paper: SR JMW.