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Title: Targeting Splicing in the Treatment of Human Disease
Author: Suñé Pou, Marc
Prieto-Sánchez, Silvia
Boyero-Corral, Sofía
Moreno Castro, Cristina
El Yousfi, Younes
Suñé i Negre, Josep M. (Josep Maria)
Hernández-Munain, Cristina
Suñé, Carlos
Keywords: Nanopartícules
Teràpia genètica
Malalties hereditàries
Gene therapy
Genetic diseases
Issue Date: 2017
Publisher: MDPI
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. Keywords: alternative splicing, precursor messenger RNA, therapy, genetic disease
Note: Reproducció del document publicat a:
It is part of: Genes, 2017, vol. 8, num. 87, p. 1-17
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ISSN: 2073-4425
Appears in Collections:Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica)

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