Ontology highlight
ABSTRACT:
SUBMITTER: Lim KH
PROVIDER: S-EPMC7347940 | biostudies-literature | 2020 Jul
REPOSITORIES: biostudies-literature
Lim Kian Huat KH Han Zhou Z Jeon Hyun Yong HY Kach Jacob J Jing Enxuan E Weyn-Vanhentenryck Sebastien S Downs Mikaela M Corrionero Anna A Oh Raymond R Scharner Juergen J Venkatesh Aditya A Ji Sophina S Liau Gene G Ticho Barry B Nash Huw H Aznarez Isabel I
Nature communications 20200709 1
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 productive mRNA. Bioinformatics analysis of RNA sequencing data identifies non-produ ...[more]