Barbon E - Search Results

1

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics.

Barbon E, Ferrarese M, van Wittenberghe L, Sanatine P, Ronzitti G, Collaud F, Colella P, Pinotti M, Mingozzi F. Barbon E, et al. Mol Ther Nucleic Acids. 2016 Nov 29;5(11):e392. doi: 10.1038/mtna.2016.97. Mol Ther Nucleic Acids. 2016. PMID: 27898092 Free PMC article.

2

Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides.

Balestra D, Barbon E, Scalet D, Cavallari N, Perrone D, Zanibellato S, Bernardi F, Pinotti M. Balestra D, et al. Among authors: barbon e. Hum Mol Genet. 2015 Sep 1;24(17):4809-16. doi: 10.1093/hmg/ddv205. Epub 2015 Jun 10. Hum Mol Genet. 2015. PMID: 26063760 Free PMC article.

3

Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein Function.

Tajnik M, Rogalska ME, Bussani E, Barbon E, Balestra D, Pinotti M, Pagani F. Tajnik M, et al. Among authors: barbon e. PLoS Genet. 2016 May 26;12(5):e1006082. doi: 10.1371/journal.pgen.1006082. eCollection 2016 May. PLoS Genet. 2016. PMID: 27227676 Free PMC article.

4

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. Sci Rep. 2016 Jun 24;6:28304. doi: 10.1038/srep28304. Sci Rep. 2016. PMID: 27341548 Free PMC article.

5

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

Lattanzi A, Duguez S, Moiani A, Izmiryan A, Barbon E, Martin S, Mamchaoui K, Mouly V, Bernardi F, Mavilio F, Bovolenta M. Lattanzi A, et al. Among authors: barbon e. Mol Ther Nucleic Acids. 2017 Jun 16;7:11-19. doi: 10.1016/j.omtn.2017.02.004. Epub 2017 Feb 10. Mol Ther Nucleic Acids. 2017. PMID: 28624187 Free PMC article.

6

Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model.

Donadon I, Pinotti M, Rajkowska K, Pianigiani G, Barbon E, Morini E, Motaln H, Rogelj B, Mingozzi F, Slaugenhaupt SA, Pagani F. Donadon I, et al. Among authors: barbon e. Hum Mol Genet. 2018 Jul 15;27(14):2466-2476. doi: 10.1093/hmg/ddy151. Hum Mol Genet. 2018. PMID: 29701768 Free PMC article.

7

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

Fitzpatrick Z, Leborgne C, Barbon E, Masat E, Ronzitti G, van Wittenberghe L, Vignaud A, Collaud F, Charles S, Simon Sola M, Jouen F, Boyer O, Mingozzi F. Fitzpatrick Z, et al. Among authors: barbon e. Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129. doi: 10.1016/j.omtm.2018.02.003. eCollection 2018 Jun 15. Mol Ther Methods Clin Dev. 2018. PMID: 29766022 Free PMC article.

8

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. Among authors: barbon e. 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

9

Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.

Le Quellec S, Dane AP, Barbon E, Bordet JC, Mingozzi F, Dargaud Y, Marais T, Biferi MG, Négrier C, Nathawani AC, Enjolras N. Le Quellec S, et al. Among authors: barbon e. Thromb Haemost. 2019 Dec;119(12):1956-1967. doi: 10.1055/s-0039-1697658. Epub 2019 Oct 28. Thromb Haemost. 2019. PMID: 31659733

10

Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors.

Barbon E, Ayme G, Mohamadi A, Ottavi JF, Kawecki C, Casari C, Verhenne S, Marmier S, van Wittenberghe L, Charles S, Collaud F, Denis CV, Christophe OD, Mingozzi F, Lenting PJ. Barbon E, et al. EMBO Mol Med. 2020 Apr 7;12(4):e11298. doi: 10.15252/emmm.201911298. Epub 2020 Mar 11. EMBO Mol Med. 2020. PMID: 32159286 Free PMC article.

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