[1] Univ Massachusetts, Sch Med, Dept Neurol, Worcester, MA USA
[2] Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA USA
[3] Johns Hopkins Sch Med, Kennedy Krieger Inst, Baltimore, VA USA
[4] Johns Hopkins Sch Med, Grad Program Cellular & Mol Med, Baltimore, VA USA
[5] Johns Hopkins Sch Med, Dept Neurol, Baltimore, VA USA
[6] Johns Hopkins Sch Med, Dept Neurosci, Baltimore, VA USA
[7] Solid Biosci LLC, Cambridge, MA USA