O.24Biodistribution of onasemnogene abeparvovec (AVXS-101) DNA, mRNA, and SMN protein in human tissue

• DOI: 10.1016/j.nmd.2019.06.307
• Publication Date: 2019-09-29T08:55:37Z
• Authors: B. Kaspar; G. Thomsen; C. Hsieh; J. Do; S. Solano; B. Chu; B. Barkho; M. Fugere; P. Kaufmann; K. Foust; A. Kaspar; J. L'Italien; D. Sproule; D. Feltner; W. Chung; A. Burghes; V. McGovern; R. Hevner; M. Conces; J. Mendell
• Keywords: 03 medical and health sciences; 0302 clinical medicine