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

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 fu...

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Veröffentlicht in:Nat Commun
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group UK 2020
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7347940/
https://ncbi.nlm.nih.gov/pubmed/32647108
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-17093-9
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