Mar 2, 2017

Targeting Splicing in the Treatment of Human Disease

Genes
Marc Suñé-PouCarlos Suñé

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 trans-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.

  • References116
  • Citations5

References

  • References116
  • Citations5

Citations

Mentioned in this Paper

Regulation of Biological Process
Gene Expression
Heterogeneous Nuclear RNA
Nuclear mRNA Cis Splicing, via Spliceosome
Trans-Splicing
RNA Splicing
RNA, Messenger
Oligonucleotides, Antisense
Alternating Hemiplegia of Childhood
Transcript

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