1The Children's Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, USA
2Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia, USA.
3Cell & Molecular Therapies, Sydney Cancer Centre, Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia
4Gene and Stem Cell Therapy Program, Centenary Institute of Cancer Medicine and Cell Biology, University of Sydney, New South Wales, Australia
5University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, Pittsburgh, USA
6University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, USA
7Present address: Bayer HealthCare LLC, 800 Dwight Way, Berkeley, California 94710, USA.,
8The Wistar Institute, Philadelphia, USA,
9Howard Hughes Medical Institute, Philadelphia, USA