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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Detalles Bibliográficos
Autores principales: Barbon, Elena, Pignani, Silvia, Branchini, Alessio, Bernardi, Francesco, Pinotti, Mirko, Bovolenta, Matteo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4920032/
https://www.ncbi.nlm.nih.gov/pubmed/27341548
http://dx.doi.org/10.1038/srep28304

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