1. Department of Medicine, Division of Pediatric Hematology Oncology, Children's Hospital Boston, Dana-Farber Cancer Institute, and Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Boston, MA;
2. Harvard Stem Cell Institute, Cambridge, MA;
3. Howard Hughes Medical Institute, Boston, MA;
4. Division of Hematology, Brigham and Women's Hospital, Boston, MA; and
5. Manton Center for Orphan Disease Research, Boston, MA