Targeting the polyadenylation signal of Pre-mRNA: A new gene silencing approach for facioscapulohumeral dystrophy - INRAE - Institut national de recherche pour l’agriculture, l’alimentation et l’environnement Access content directly
Journal Articles (Review Article) International Journal of Molecular Sciences Year : 2018

Targeting the polyadenylation signal of Pre-mRNA: A new gene silencing approach for facioscapulohumeral dystrophy

Abstract

Facioscapulohumeral dystrophy (FSHD) is characterized by the contraction of the D4Z4 array located in the sub-telomeric region of the chromosome 4, leading to the aberrant expression of the DUX4 transcription factor and the mis-regulation of hundreds of genes. Several therapeutic strategies have been proposed among which the possibility to target the polyadenylation signal to silence the causative gene of the disease. Indeed, defects in mRNA polyadenylation leads to an alteration of the transcription termination, a disruption of mRNA transport from the nucleus to the cytoplasm decreasing the mRNA stability and translation efficiency. This review discusses the polyadenylation mechanisms, why alternative polyadenylation impacts gene expression, and how targeting polyadenylation signal may be a potential therapeutic approach for FSHD.
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hal-02624975 , version 1 (26-05-2020)

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Anne Charlotte Marsollier, Romain Joubert, Virginie Mariot, Julie Dumonceaux. Targeting the polyadenylation signal of Pre-mRNA: A new gene silencing approach for facioscapulohumeral dystrophy. International Journal of Molecular Sciences, 2018, 19 (5), ⟨10.3390/ijms19051347⟩. ⟨hal-02624975⟩
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