Targeting Splicing in the Treatment of Human Disease

Overview

Journal Genes (Basel)

Publisher MDPI

Date 2017 Mar 2

PMID 28245575

Citations 22

Authors

Marc Sune-Pou

Silvia Prieto-Sanchez

Sofia Boyero-Corral

Cristina Moreno-Castro

Younes El Yousfi

Josep Ma Sune-Negre

Cristina Hernandez-Munain

Carlos Sune

Affiliations

Soon will be listed here.

Abstract

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 tools, including antisense oligonucleotides and -splicing, have been developed to target and alter splicing to correct misregulated gene expression or to modulate transcript isoform levels. At present, deregulated AS is recognized as an important area for therapeutic intervention. Here, we summarize the major hallmarks of the splicing process, the clinical implications that arise from alterations in this process, and the current tools that can be used to deliver, target, and correct deficiencies of this key pre-mRNA processing event.

Citing Articles

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