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Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy.
Haematologica. 2021 Jun 1;106(6):1624-1635. doi: 10.3324/haematol.2019.239202.
Haematologica. 2021.
PMID: 32467137
Free PMC article.
Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts.
Pignani S, Zappaterra F, Barbon E, Follenzi A, Bovolenta M, Bernardi F, Branchini A, Pinotti M.
Pignani S, et al.
Biochim Biophys Acta Gene Regul Mech. 2019 Jun;1862(6):619-624. doi: 10.1016/j.bbagrm.2019.04.002. Epub 2019 Apr 18.
Biochim Biophys Acta Gene Regul Mech. 2019.
PMID: 31005673
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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.
Barbon E, Pignani S, Branchini A, Bernardi F, Pinotti M, Bovolenta M.
Barbon E, et al. Among authors: pignani s.
Sci Rep. 2016 Jun 24;6:28304. doi: 10.1038/srep28304.
Sci Rep. 2016.
PMID: 27341548
Free PMC article.
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The carboxyl-terminal region of human coagulation factor X as a natural linker for fusion strategies.
Ferrarese M, Pignani S, Lombardi S, Balestra D, Bernardi F, Pinotti M, Branchini A.
Ferrarese M, et al. Among authors: pignani s.
Thromb Res. 2019 Jan;173:4-11. doi: 10.1016/j.thromres.2018.11.007. Epub 2018 Nov 9.
Thromb Res. 2019.
PMID: 30453126
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The chaperone-like sodium phenylbutyrate improves factor IX intracellular trafficking and activity impaired by the frequent p.R294Q mutation.
Pignani S, Todaro A, Ferrarese M, Marchi S, Lombardi S, Balestra D, Pinton P, Bernardi F, Pinotti M, Branchini A.
Pignani S, et al.
J Thromb Haemost. 2018 Oct;16(10):2035-2043. doi: 10.1111/jth.14236. Epub 2018 Aug 9.
J Thromb Haemost. 2018.
PMID: 29993188
Free article.
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