[ES] イブニングセミナー

[ES-1] AVXS—101 single—dose gene—replacement therapy for spinal muscular atrophy

Douglas M. Sproule¹、Jerry R. Mendell^2、3、4、Samiah Al-Zaidy^2、3、Richard Shell³、W. Dave Arnold⁴、Louise Rodino-Klapac^2、4、Thomas W. Prior⁵、Linda Lowes^2、3、Lindsay Alfano^2、3、Katherine Berry^2、3、Kathleen Church^2、3、John T. Kissel³、Sukumar Nagendran¹、James L'Italien¹、Courtney Wells¹、Arthur H.M. Burghes^4、6、Kevin D. Foust¹、Kathrin Meyer²、Shibi Likhite²、Brian K. Kaspar^{2、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)