Targeting splicing in the treatment of human disease

M Suñé-Pou, S Prieto-Sánchez, S Boyero-Corral… - Genes, 2017 - mdpi.com
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 …

[引用][C] Targeting splicing in the treatment of human disease. Genes. 2017

M Suñé-Pou, S Prieto-Sánchez, S Boyero-Corral…
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