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. 2019 Jun 21;20(12):3036. doi: 10.3390/ijms20123036

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© 2019 by the authors.

Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).

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Gene editing and transcription modulation-based approaches for coagulation factor deficiencies. Approaches aimed at correcting and/or modulating the expression of coagulation factor genes through ZFNs (A), cleavage by CRISPR/Cas9 (B), engineering of the albumin locus to drive F9 gene expression (C), correction of intron 22 inversion in F8 gene (D), and TALE-TF or the CRISPRa systems leading to an increase in luciferase (reporter constructs) or endogenous activity due to F7 promoter transactivation (E). Asterisks represent the c.-94C>G (*) and c.-61T>G (**) nucleotide changes.