About the Authors

Thierry Vilboux

Affiliation Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America

Carla Ciccone

Affiliation Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America

Jan K. Blancato

Affiliation Department of Oncology, Georgetown University Medical Center, Washington, D.C., United States of America

Gerald F. Cox

Affiliations Division of Genetics, Department of Pediatrics, Harvard Medical School, Children's Hospital Boston, Boston, Massachusetts, United States of America, Genzyme Corporation, Cambridge, Massachusetts, United States of America

Charu Deshpande

Affiliation Department of Genetics, Guy's Hospital, London, United Kingdom

Wendy J. Introne

Affiliation Office of the Clinical Director, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America

William A. Gahl

Affiliations Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America, Office of the Clinical Director, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America

Ann C. M. Smith

Affiliation Office of the Clinical Director, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America

Marjan Huizing

mhuizing@mail.nih.gov

Affiliation Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, United States of America

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

Conceived and designed the experiments: TV ACMS MH. Performed the experiments: TV JKB CC. Analyzed the data: TV CC ACMS MH. Wrote the paper: TV WAG ACMS MH. Recruited patients and performed clinical investigations of the patients: GFC CD WJI WAG ACMS.