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

Authors :
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
Source :
Nature Communications, Vol 11, Iss 1, Pp 1-13 (2020)
Publication Year :
2020
Publisher :
Nature Portfolio, 2020.

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.

Subjects

Subjects :
Science

Details

Language :
English
ISSN :
20411723
Volume :
11
Issue :
1
Database :
Directory of Open Access Journals
Journal :
Nature Communications
Publication Type :
Academic Journal
Accession number :
edsdoj.b4b17242c1b24dc9905cc03dd97077f2
Document Type :
article
Full Text :
https://doi.org/10.1038/s41467-020-17093-9