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Targeting the Polyadenylation Signal of Pre-mRNA: A New Gene Silencing Approach for Facioscapulohumeral Dystrophy

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 bee...

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Vydáno v:Int J Mol Sci
Hlavní autoři: Marsollier, Anne-Charlotte, Joubert, Romain, Mariot, Virginie, Dumonceaux, Julie
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2018
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5983732/
https://ncbi.nlm.nih.gov/pubmed/29751519
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms19051347
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