Utrophin up-regulation by artificial transcription factors induces muscle rescue and impacts the neuromuscular junction in mdx mice

Up-regulation of the dystrophin-related gene utrophin represents a promising therapeutic strategy for the treatment of Duchenne Muscular Dystrophy (DMD). In order to re-program the utrophin expression level in muscle, we engineered artificial zinc finger transcription factors (ZF-ATFs) that target t...

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Udgivet i:Biochim Biophys Acta
Main Authors: Pisani, Cinzia, Strimpakos, Georgios, Gabanella, Francesca, Di Certo, Maria Grazia, Onori, Annalisa, Severini, Cinzia, Luvisetto, Siro, Farioli-Vecchioli, Stefano, Carrozzo, Irene, Esposito, Antonio, Canu, Tamara, Mattei, Elisabetta, Corbi, Nicoletta, Passananti, Claudio
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier Pub. Co 2018
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5851675/
https://ncbi.nlm.nih.gov/pubmed/29408646
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbadis.2018.01.030
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