[1] Boston Childrens Hosp, Div Hematol Oncol, Boston, MA USA
[2] Boston Childrens Hosp, Stem Cell Program, Boston, MA USA
[3] Dana Farber Canc Inst, Pediat Oncol, Boston, MA USA
[4] Harvard Stem Cell Inst, Harvard Initiat RNA Med, Boston, MA USA
[5] Harvard Med Sch, Dept Pediat, Boston, MA USA
[6] Edmond & Lily Safra Childrens Hosp, Sheba Med Ctr, Tel Hashomer, Israel
[7] Tel Aviv Univ, Sackler Sch Med, Tel Aviv, Israel
[8] Duke Univ, Div Pediat & Cellular Therapy, Div Cellular Therapy, Med Ctr, Durham, NC USA
[9] Cleveland Clin, Ctr Pediat Neurosci, Mitochondrial Med, Cleveland, OH USA
[10] Boston Childrens Hosp, Harvard Med Sch, Dept Pathol, Boston, MA USA
[11] Harvard Med Sch, Dept Pediat, Boston, MA 02115 USA