Article
Global identification of hnRNP A1 binding sites for SSO-based splicing modulation.
BMC biology - 5 Jul 2016
Bruun Gitte H, Doktor Thomas K, Borch-Jensen Jonas, Masuda Akio, Krainer Adrian R, Ohno Kinji, Andresen Brage S
Abstract excerpt
BACKGROUND: Many pathogenic genetic variants have been shown to disrupt mRNA splicing. Besides splice mutations in the well-conserved splice sites, mutations in splicing regulatory elements (SREs) may deregulate splicing and cause disease. A promising therapeutic approach is to compensate for this deregulation by blocking other SREs with splice-switching oligonucleotides (SSOs). However, the location and sequence...
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