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An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes

Tailored approaches to restore defective transcription responsible for severe diseases have been poorly explored. We tested transcription activator-like effectors fused to an activation domain (TALE-TFs) in a coagulation factor VII (FVII) deficiency model. In this model, the deficiency is caused by...

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Detaylı Bibliyografya
Yayımlandı:Sci Rep
Asıl Yazarlar: Barbon, Elena, Pignani, Silvia, Branchini, Alessio, Bernardi, Francesco, Pinotti, Mirko, Bovolenta, Matteo
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2016
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4920032/
https://ncbi.nlm.nih.gov/pubmed/27341548
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep28304
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