Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor

by Juergen Scharner, Sabrina Qi, Frank Rigo, C. Frank Bennett, Adrian R. Krainer
Published 2019-06-01

Uncoordinated transcription and compromised muscle function in the lmna-null mouse model of Emery- Emery-Dreyfuss muscular dystrophy.

by Viola F Gnocchi, Juergen Scharner, Zhe Huang, Ken Brady, Jaclyn S Lee, Robert B White, Jennifer E Morgan, Yin-Biao Sun, Juliet A Ellis, Peter S Zammit
Published 2011-02-01

Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

by Kian Huat Lim, Zhou Han, Hyun Yong Jeon, Jacob Kach, Enxuan Jing, Sebastien Weyn-Vanhentenryck, Mikaela Downs, Anna Corrionero, Raymond Oh, Juergen Scharner, Aditya Venkatesh, Sophina Ji, Gene Liau, Barry Ticho, Huw Nash, Isabel Aznarez
Published 2020-07-01