Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice

by Hichem Tasfaout, Suzie Buono, Shuling Guo, Christine Kretz, Nadia Messaddeq, Sheri Booten, Sarah Greenlee, Brett P. Monia, Belinda S. Cowling, Jocelyn Laporte
Published 2017-06-01

Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys

by Colby S Shemesh, Rosie Z Yu, Hans J Gaus, Sarah Greenlee, Noah Post, Karsten Schmidt, Michael T Migawa, Punit P Seth, Thomas A Zanardi, Thazha P Prakash, Eric E Swayze, Scott P Henry, Yanfeng Wang
Published 2016-01-01