Article
Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression.
Nature communications - 9 Jul 2020
Lim Kian Huat, Han Zhou, Jeon Hyun Yong, Kach Jacob, Jing Enxuan, Weyn-Vanhentenryck Sebastien, Downs Mikaela, Corrionero Anna, Oh Raymond, Scharner Juergen, Venkatesh Aditya, Ji Sophina, Liau Gene, Ticho Barry, Nash Huw, Aznarez Isabel
Abstract excerpt
While most monogenic diseases are caused by loss or reduction of protein function, the need for technologies that can selectively increase levels of protein in native tissues remains. Here we demonstrate that antisense-mediated modulation of pre-mRNA splicing can increase endogenous expression of full-length protein by preventing naturally occurring non-productive alternative splicing and promoting generation of...
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