Article
An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes.
Scientific reports - 24 Jun 2016
Barbon Elena, Pignani Silvia, Branchini Alessio, Bernardi Francesco, Pinotti Mirko, Bovolenta Matteo
Abstract excerpt
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 the -94C > G or -61T > G mutation, which abrogate the binding of Sp1 or HNF-4 transcription factors. Reporter assays...
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