血小板定向基因治疗克服了针对凝血因子VIII的抑制性抗体。 - Suppr

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超能文献

Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.

作者信息

Chuah M K, Vandendriessche T

机构信息

Department of Gene Therapy and Regenerative Medicine, Free University of Brussels (VUB), Brussels, Belgium.

出版信息

J Thromb Haemost. 2012 Aug;10(8):1566-9. doi: 10.1111/j.1538-7836.2012.04794.x.

DOI:10.1111/j.1538-7836.2012.04794.x

PMID:22642298

Abstract

摘要

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Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.

作者信息

Chuah M K, Vandendriessche T

机构信息

Department of Gene Therapy and Regenerative Medicine, Free University of Brussels (VUB), Brussels, Belgium.

出版信息

J Thromb Haemost. 2012 Aug;10(8):1566-9. doi: 10.1111/j.1538-7836.2012.04794.x.

DOI:10.1111/j.1538-7836.2012.04794.x

PMID:22642298

Abstract

摘要

相似文献

Platelet-directed gene therapy overcomes inhibitory antibodies to factor VIII.

J Thromb Haemost. 2012 Aug;10(8):1566-9. doi: 10.1111/j.1538-7836.2012.04794.x.

Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.

J Thromb Haemost. 2012 Aug;10(8):1570-80. doi: 10.1111/j.1538-7836.2012.04791.x.

Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.

Mol Ther. 2015 Apr;23(4):617-26. doi: 10.1038/mt.2015.20. Epub 2015 Feb 6.

The impact of GPIbα on platelet-targeted FVIII gene therapy in hemophilia A mice with pre-existing anti-FVIII immunity.

J Thromb Haemost. 2019 Mar;17(3):449-459. doi: 10.1111/jth.14379. Epub 2019 Feb 3.

Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A.

J Thromb Haemost. 2007 Feb;5(2):352-61. doi: 10.1111/j.1538-7836.2007.02346.x.

A shot in the bone corrects a genetic disease.

Mol Ther. 2015 Apr;23(4):614-5. doi: 10.1038/mt.2015.38.

Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells.

Blood. 2014 Jan 16;123(3):395-403. doi: 10.1182/blood-2013-08-520478. Epub 2013 Nov 22.

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity.

Blood. 2008 Oct 1;112(7):2713-21. doi: 10.1182/blood-2008-02-138214. Epub 2008 May 21.

Immune tolerance induced by platelet-targeted factor VIII gene therapy in hemophilia A mice is CD4 T cell mediated.

J Thromb Haemost. 2017 Oct;15(10):1994-2004. doi: 10.1111/jth.13800. Epub 2017 Sep 11.

Phenotypic correction of hemophilia A by ectopic expression of activated factor VII in platelets.

Mol Ther. 2008 Aug;16(8):1359-65. doi: 10.1038/mt.2008.117. Epub 2008 Jun 3.