1. Stem Cell Transplantation Program, Division of Pediatric Hematology/Oncology, Manton Center for Orphan Disease Research, Howard Hughes Medical Institute, Boston Children's Hospital and Dana Farber Cancer Institute;
2. Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School;
3. Harvard Stem Cell Institute; Boston, Massachusetts 02115;
4. Division of Hematology, Brigham and Women's Hospital;
5. Broad Institute;