1. Stem Cell Program, Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02115, USA
2. Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Boston, MA 02115, USA
3. Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA
4. Harvard Initiative for RNA Medicine, Boston, MA 02115, USA
5. Harvard Stem Cell Institute, Cambridge, MA 02138, USA