A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.
about
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical applicationEndothelial cell control of thrombosisHemophilia A: an ideal disease to correct in uteroProgress and challenges in the development of a cell-based therapy for hemophilia AComplexity and diversity of F8 genetic variations in the 1000 genomesGene therapy for hemophiliaGene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.FcRn Rescues Recombinant Factor VIII Fc Fusion Protein from a VWF Independent FVIII Clearance Pathway in Mouse Hepatocytes.Changes in Dietary Fat Content Rapidly Alters the Mouse Plasma Coagulation Profile without Affecting Relative Transcript Levels of Coagulation FactorsFactor VIII Is Synthesized in Human Endothelial Cells, Packaged in Weibel-Palade Bodies and Secreted Bound to ULVWF Strings.Donor antigen-primed regulatory T cells permit liver regeneration and phenotype correction in hemophilia A mouse by allogeneic bone marrow stem cells.Association of Single Nucleotide Polymorphisms in the ST3GAL4 Gene with VWF Antigen and Factor VIII Activity.In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCsCharacterization of a genetically engineered mouse model of hemophilia A with complete deletion of the F8 gene.Patterns of expression of factor VIII and von Willebrand factor by endothelial cell subsets in vivo.Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models.Missense mutations near the N-glycosylation site of the A2 domain lead to various intracellular trafficking defects in coagulation factor VIII.Obstacles and future of gene therapy for hemophilia.Evidence That Factor VIII Forms a Bivalent Complex with the Low Density Lipoprotein (LDL) Receptor-related Protein 1 (LRP1): IDENTIFICATION OF CLUSTER IV ON LRP1 AS THE MAJOR BINDING SITE.Gene therapy for immune tolerance induction in hemophilia with inhibitors.Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A.Potential for cellular stress response to hepatic factor VIII expression from AAV vectorHigh-affinity von Willebrand factor binding does not affect the anatomical or hepatocellular distribution of factor VIII in rats.Mice deficient in the anti-haemophilic coagulation factor VIII show increased von Willebrand factor plasma levels.Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A.Native plasma-derived FVIII/VWF complex has lower sensitivity to FVIII inhibitors than the combination of isolated FVIII and VWF proteins. Impact on Bethesda assay titration of FVIII inhibitors.Factor VIII/V C-domain swaps reveal discrete C-domain roles in factor VIII function and intracellular trafficking.The search for the origin of factor VIII synthesis and its impact on therapeutic strategies for hemophilia A.Hemostatic Factors, APOL1, and ESRD Risk: Another Piece of the Puzzle?Nogo-B receptor deficiency increases liver X receptor alpha nuclear translocation and hepatic lipogenesis through an adenosine monophosphate-activated protein kinase alpha-dependent pathway.Comparison of platelet-derived and plasma factor VIII efficacy using a novel native whole blood thrombin generation assay.Targeting factor VIII expression to platelets for hemophilia A gene therapy does not induce an apparent thrombotic risk in mice.Desmopressin in moderate hemophilia A patients: a treatment worth considering.Platelets are a safe way to deliver factor VIII. After 13 years of preclinical research it is now time for a clinical trial.Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9.Evaluation of high-fat high-fructose diet treatment in factor VIII (coagulation factor)-deficient mouse model.
P2860
Q26748949-71CE6D1C-24B0-4779-9C49-4BD54A3B4360Q26781779-01070C38-B126-42CC-889E-C32CFCFE8E28Q26852638-BD5DF0D7-D756-40BE-889E-05EE1ADE89AFQ27027298-4F328DC9-DB58-411D-B74C-BBEAB0969F2FQ27347944-8364F6FF-BFFC-4A0B-A08A-3ED606F0F713Q28081911-C536E899-3248-4E29-A5E1-D718A1EB1C98Q33881920-1B51F6B1-9F01-4AF1-9ABC-3775F2314C49Q35612397-81208C60-E46E-4151-A246-20C5F8AF602BQ35691994-AC4C759A-EA9C-4A1B-AFF7-9AB78457E9FCQ35810108-2819EC60-0FDF-48B9-81F2-50555D1FB838Q35885551-435DE6E0-4339-4CFA-A4D7-56E8A2AC7885Q36120096-D907F8BE-F871-4AAC-9805-9FE9B698B288Q36442862-D0E43E68-9A19-4F75-AC6A-C8889E19C632Q36586264-5F95CEFD-C935-4640-A65B-A3EAAE6D4054Q37076404-18D27AF6-4F25-4756-8B95-8F87A8ADCECAQ37292357-DADFA44A-EC44-4993-827E-4937AC5366F3Q37316242-322951FE-E698-4B6E-B4A9-48C308CBAF5AQ37715039-704CE2A9-F91B-4084-A4C2-9D42EE327283Q38529941-A1ACA5EB-7897-40E3-B712-E19AE890D401Q38735281-0E10DC3C-D497-428E-9A79-984231CB1350Q38803022-18EC62CF-6582-4A66-88C6-4485A47D84F0Q38944642-CA62EB17-2ABB-4FB0-B427-8BE5885EAF7DQ39293496-75A32CE2-DB2B-49BB-AF74-BBD6D92C6AA6Q39631688-86FC4C59-E3C7-48E3-A536-AE6B7882F87FQ41512070-468E799A-06C0-4671-8C59-A070B40009F9Q41580780-E42C242F-7594-486C-AEBF-D59DE0279766Q41809588-BE6090D7-A4A8-4651-AC2B-A8316232F8C2Q41861345-3DAFC976-38C6-4366-AA6D-686FED5D2612Q42384645-4271EE78-300F-49BF-8320-CE6512B52F77Q42569055-321D8567-3459-4BE1-A6D4-522018867A84Q42674175-7F91DFC2-7E32-4B34-BD43-59839C7D2294Q42870426-83D412A1-E59D-4DBF-A675-B4664131D3DDQ45857189-A97061DC-8C31-4C81-8A0B-EADB337D7D78Q47203639-C3C39726-73CD-4157-A974-86EEA2AA57FAQ51769404-A830C22A-06C8-47FC-8F42-9785F902D999Q52431082-47CC4C80-2F28-4493-A022-725CE915E7BAQ52587797-E3C89C56-AC3F-4DAC-B0FE-C9073CA56906
P2860
A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.
description
2014 nî lūn-bûn
@nan
2014 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
A conditional knockout mouse m ...... source of plasma factor VIII.
@ast
A conditional knockout mouse m ...... source of plasma factor VIII.
@en
type
label
A conditional knockout mouse m ...... source of plasma factor VIII.
@ast
A conditional knockout mouse m ...... source of plasma factor VIII.
@en
prefLabel
A conditional knockout mouse m ...... source of plasma factor VIII.
@ast
A conditional knockout mouse m ...... source of plasma factor VIII.
@en
P2093
P2860
P1433
P1476
A conditional knockout mouse m ...... source of plasma factor VIII.
@en
P2093
Hartmut Weiler
Matthew T Hille
Qizhen Shi
Robert R Montgomery
Scot A Fahs
P2860
P304
P356
10.1182/BLOOD-2014-02-555151
P407
P577
2014-04-04T00:00:00Z