1. Division of Hematology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, PA;
2. Division of Genetics and Cell Biology, San Raffaele Scientific Institute, Milan, Italy;
3. Vita-Salute San Raffaele University, Milan, Italy;
4. Ionis Pharmaceuticals, Inc, Carlsbad, CA;
5. San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;
6. Aevi Genomic Medicine, Inc, Wayne, PA;
7. Medgenics Medical Israel, Ltd, Misgav Dov, Israel;
8. Department of Basic Science and Craniofacial Biology, New York University College of Dentistry, New York, NY;
9. Perelman School of Medicine and
10. Cell and Molecular Biology Affinity Group, University of Pennsylvania, Philadelphia, PA; and
11. Raymond G. Perelman Center for Cellular and Molecular Therapeutics, and
12. Penn Center for Musculoskeletal Disorders, Children’s Hospital of Philadelphia, Philadelphia, PA