A method of using a vector containing a nucleic acid encoding a FVIII protein and a nucleic acid variant is disclosed. In certain embodiments, the method of treating a human with hemophilia A encodes a factor VIII (FVIII) (hFVIII-BDD) having a nucleic acid encoding factor VIII (FVIII) or...
factor VIII n. A protein substance in blood plasma that is an essential part of the blood-clotting process. Most cases of hemophilia are caused by a genetic defect that leads to a deficiency of this factor. Also calledantihemophilic factor. ...
High-level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors. Two liver-specific nonviral gene transfer vectors have been developed to accommodate heterologous genes. The expression cassettes contain (1) a hepatic loc... H Carol,Miao,Xin,... - 《Human...
It may also provide insights into the benefits of platelet FVIII storage before attempting permanent pFVIII gene therapy to demonstrate efficacy in the presence of a patient's particular inhibitors and specific target organs. It may also be a useful adjuvant for bleeding challenges after marrow ...
Factor VIII Gene This gene is in band Xq28, 1.5Mb from the telomere. It spans 186kb, contains 26 exons, and is oriented so that the promoter lies telomeric to the rest of the gene. A CpG island in intron 22 of thefactor VIIIgene is the origin of two nested genes: F8A and F8B. ...
One repeat is located in intron 1 (int1h-1) of the F8 gene and the other is located 140 kb upstream (int1h-2), outside the F8 gene. This inversion displaces exon 1 roughly 140 kb upstream of the F8 gene and fully disrupts Factor VIII expression18,19. Restoring 2–5% of the...
Hemophilia A, which results in defective or deficient factor VIII (FVIII) protein, is one of the genetic diseases that has been addressed through gene therapy trials. FVIII synthesis does not occur in normal megakaryocytes. In hemophilia patients who have inhibitors to FVIII activity, megakaryocyte...
Ex Vivo Gene Therapy for Hemophilia A That Enhances Safe Delivery and Sustained In Vivo Factor VIII Expression from Lentivirally Engineered Endothelial Progenitors 来自 Wiley 喜欢 0 阅读量: 34 作者: H Matsui,M Shibata,B Brown,A Labelle,C Hegadorn,C Andrews,RP Hebbel,J Galipeau,C Hough,...
Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A 来自 ResearchGate 喜欢 0 阅读量: 34 作者:CL Eckhardt,ASV Velzen,M Peters 展开 DOI: 10.1182/blood-2014-04-567339 年份: 2014 收藏 引用 批量引用 报错 分享 全部来源 免费下载 求助全文 Semantic Scholar (...
Hemophilia A in its severe form is a life-threatening hemorrhagic disease that is caused by mutations in the factor VIII (FVIII) gene (symbol F8). About 25% of patients who receive replacement therapy develop neutralizing antibodies that inhibit the function of substituted FVIII. Long-term applica...