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Title

Targeting Splicing in the Treatment of Human Disease

AuthorsSuñé-Pou, Marc; Prieto-Sánchez, Silvia; Boyero-Corral, Sofía; Moreno-Castro, Cristina; El Yousfi, Younes; Suñé-Negre, Josep Mª; Hernández-Munaín, Cristina; Suñé, Carlos
Issue Date24-Feb-2017
PublisherMultidisciplinary Digital Publishing Institute
CitationGenes 8 (3): 87 (2017)
AbstractThe 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 <i>trans</i>-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.
Publisher version (URL)https://doi.org/10.3390/genes8030087
URIhttp://hdl.handle.net/10261/149356
DOI10.3390/genes8030087
Identifiersdoi: 10.3390/genes8030087
Appears in Collections:(IPBLN) Artículos
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