Douglas M. Sproule1、Jerry R. Mendell2、3、4、Samiah Al-Zaidy2、3、Richard Shell3、W. Dave Arnold4、Louise Rodino-Klapac2、4、Thomas W. Prior5、Linda Lowes2、3、Lindsay Alfano2、3、Katherine Berry2、3、Kathleen Church2、3、John T. Kissel3、Sukumar Nagendran1、James L'Italien1、Courtney Wells1、Arthur H.M. Burghes4、6、Kevin D. Foust1、Kathrin Meyer2、Shibi Likhite2、Brian K. Kaspar2、3、4、1 (AveXis, Inc., Bannockburn, IL, USA1、Center for Gene Therapy Nationwide Children's Hospital, Columbus, OH, USA2、Department of Pediatrics, Ohio State University, Columbus, OH, USA3、Department of Neurology, Ohio State University, Columbus, OH, USA4、Department of Pathology, Ohio State University, Columbus, OH, USA5、Department of Molecular and Cellular Biochemistry, Ohio State University, Columbus, OH, USA6)