Methods of using vvectors comprising nucleic acid and nucleic acid variants encoding FVIII protein are disclosed. In particular embodiments, a method of treating a human having hemophilia A includes administering a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid encoding ...
1.Srivastava A, Santagostino E, Dougall A, Kitchen S, Sutherland M, Pipe SW, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26 Suppl 6:1-158. 2.Nathwani AC. Gene therapy for hemophi...
(B-domaimdeletedFVIII)posttranslationallybyproteintrans-splicingaftertransferofsplitBDD-FVIIIgenebyadual-vectorsystem.InthisstudyweinvestigatedtheeffectofvWFonsecretionandactivityofintein-ligatedBDD-FVIII.Weobservedthelevelsoffull-lengthBDD-FVIIIantigensecretedintoculturesupernatantbyELISAandtheiractivitybyCoatestassay...
在肝中活跃的启动子的实例是ttr启动子,人α1-抗胰蛋白酶(haat)启动子;白蛋白,miyatake等人,j.virol.,71:5124-32(1997);乙型肝炎病毒核心启动子,sandig等人,genether.3:1002-9(1996);α-甲胎蛋白(afp),arbuthnot等人,hum.gene.ther.,7:1503-14(1996)]等。在肝脏中活跃的增强子的实例是载脂蛋白e(apoe)...
具有严重征状的患者(占血友病患者的大约一半)一个月几次地表现出出血征状(如果他们不接受以后描述的预防性替代疗法),并且该频率与中等症状的患者和轻度症状的患者的频率相比明显更高。 除了血友病和获得性血友病以外,已知由von Willebrand因子(vWF)的功能异常或缺乏造成的血管性血友病(von Willebrand′s disease)是...
4)Coagulation factor VIII gene凝血因子VIII基因 5)FVIII antibodiesFVIII抗体 6)VIII-RFVIII因子 英文短句/例句 1.Study on the effect of two methods on the preparation of VIII factor and fibrinogen两种方法制备冷沉淀对VIII因子、纤维蛋白原影响
·mL −1 , respectively, greater than that of non-vWF co-transfected cell (110 ± 18 ng·mL −1 and 1.10 ± 0.15 u·mL −1 ) or just BDD-FVIII gene transfected control cell (131 ± 25 ng·mL −1 and 1.22 ± 0.18 u·mL −1 ) indicating the benefit of vWF gene co-...
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12月7日,辉瑞和基因治疗公司Sangamo Therapeutics在第62届美国血液学学会(ASH)年会上共同宣布了针对严重血友病A患者的研究性基因疗法Giroocotogene fitelparvovec(又称SB-525或PF-07055480)1/2期临床研究Alta的最新随访数据,包括最长治疗时间85周的患者数据。结果显示,在接受该基因疗法高剂量3x1013vg/kg队列中的5名...
expression cassette with the B-domain deleted form ofFVIII(BDD-FVIII) and the EF1α promoter for insertion at exon 1 of theFVIIIlocus. Importantly, insertion of theFVIIIgene resulted in the production of a functionally active FVIII protein from the gene-corrected iPSC line-derived endothelial ...