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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Publicat a:Nat Commun
Autors principals: 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
Idioma:Inglês
Publicat: Nature Publishing Group UK 2020
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Article
Accés en línia: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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