Please use this identifier to cite or link to this item:
http://hdl.handle.net/2445/173991
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 Nanotecnologia Nanoparticles Gene therapy Genetic diseases Nanotechnology |
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: https://doi.org/10.3390/genes8030087 |
It is part of: | Genes, 2017, vol. 8, num. 87, p. 1-17 |
URI: | http://hdl.handle.net/2445/173991 |
Related resource: | https://doi.org/10.3390/genes8030087 |
ISSN: | 2073-4425 |
Appears in Collections: | Articles publicats en revistes (Farmàcia, Tecnologia Farmacèutica i Fisicoquímica) |
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