1. Division of Genetics and Genomics at Boston Children's Hospital, Boston, MA, USA
2. Department of Pediatrics and Genetics at Harvard Medical School, Boston, MA, USA
3. The Stem Cell Program at Boston Children's Hospital, Boston, MA, USA
4. The Manton Center for Orphan Disease Research at Boston Children's Hospital, Boston, MA, USA
5. Harvard Stem Cell Institute, Cambridge, MA, USA