1Division of Genetics and Genomics, Manton Center for Orphan Disease, Boston Children’s Hospital, Boston, MA, USA; Department of Pediatrics, Harvard Medical School, Boston, MA, USA; and Broad Institute of MIT and Harvard, Cambridge, MA, USA
2Howard Hughes Medical Institute, Boston Children’s Hospital, Boston, MA, USA and Department of Neurology, Harvard Medical School, Boston, MA, USA
4Department of Cell, Molecular, and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, USA
