[1] Univ Minnesota, Dept Pediat, Minneapolis, MN 55104 USA
[2] Univ Maryland, Sch Med, Dept Physiol, Baltimore, MD 21201 USA
[3] UCL, Great Ormond St Inst Child Hlth, NIHR Biomed Res Ctr, London WC1N, England
[4] Great Ormond St Hosp NHS Trust, London WC1N 1EH, England
[5] Ohio State Univ, Dept Pediat, Columbus, OH 43205 USA
[6] Nationwide Childrens Hosp, Abigail Wexner Res Inst, Ctr Gene Therapy, Columbus, OH 43205 USA
[7] Leiden Univ, Med Ctr, Dept Human Genet, Leiden, Netherlands
[8] St Louis Univ, Edward A Doisy Dept Biochem & Mol Biol, St Louis, MO 63104 USA
[9] Kennedy Krieger Inst, Ctr Genet Muscle Disorders, Baltimore, MD 21205 USA
[10] Johns Hopkins Sch Med, Dept Neurol & Neurosci, Baltimore, MD 21205 USA
[11] Radboud Univ Nijmegen, Med Ctr, Dept Neurol, Nijmegen, Netherlands
[12] George Washington Univ, Dept Genom & Precis Med, Washington, DC 20052 USA
[13] Childrens Natl Res Inst, Ctr Genet Med Res, Washington, DC 20010 USA