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Article

Targeting Splicing in the Treatment of Human Disease

24 Feb 2017

Abstract excerpt

The tightly regulated process of precursor messenger RNA (pre-mRNA) alternative splicing (AS) is a key mechanism in the regulation of gene expression. Defects in this regulatory process affect cellular functions and are the cause of many human diseases. Recent advances in our understanding of splicing regulation have led to the development of new tools for manipulating splicing for therapeutic purposes. Several...

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PMID: 28245575
DOI: 10.3390/genes8030087

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