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

Author

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, and Isabel Aznarez

Subjects
Science
Abstract

Restoration of normal gene expression is one way to treat monogenic disorders. Here the authors target naturally occurring non-productive alternative splicing using antisense oligonucleotides to promote the production of functional proteins.

Published
2020
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