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

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.

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Auteurs principaux: 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
Format: article
Langue:EN
Publié: Nature Portfolio 2020
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Accès en ligne:https://doaj.org/article/b4b17242c1b24dc9905cc03dd97077f2
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