Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. - Wikidata - wikimedia - TriplyDB

Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.

Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.

http://www.wikidata.org/entity/Q41664760

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In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application Megakaryocyte- and megakaryocyte precursor-related gene therapies The gene therapy journey for hemophilia: are we there yet?Hemophilia A: an ideal disease to correct in utero Animal models of hemophilia Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors Gene therapy for hemophilia Lessons Learned from Animal Models of Inherited Bleeding Disorders.Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors.Correction of murine Bernard-Soulier syndrome by lentivirus-mediated gene therapy.Delivery of factor VIII gene into skeletal muscle cells using lentiviral vector.In vivo efficacy of platelet-delivered, high specific activity factor VIII variants A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.Advancements in gene transfer-based therapy for hemophilia A.In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.Targeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia A.Platelets as delivery systems for disease treatments Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.Apoptotic effects of platelet factor VIII on megakaryopoiesis: implications for a modified human FVIII for platelet-based gene therapy.Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.The important role of von Willebrand factor in platelet-derived FVIII gene therapy for murine hemophilia A in the presence of inhibitory antibodies.Intravascular recovery of VWF and FVIII following intraperitoneal injection and differences from intravenous and subcutaneous injection in mice.Contribution of platelet vs. endothelial VWF to platelet adhesion and hemostasis.Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions The immunogenicity of platelet-derived FVIII in hemophilia A mice with or without preexisting anti-FVIII immunity Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment.Platelets in lung biology.Factor VIII inhibitors: von Willebrand factor makes a difference in vitro and in vivo Gene therapy approaches for stem cell protection.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 Meeting report: ninth and tenth workshops of the European Paediatric Network for Haemophilia Management (PedNet).Structure-function and regulation of ADAMTS-13 protease.In vivo models of haemophilia - status on current knowledge of clinical phenotypes and therapeutic interventions.Storage and regulated secretion of factor VIII in blood outgrowth endothelial cells Back to the future: a recent history of haemophilia treatment.

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P2860

Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.

http://www.wikidata.org/entity/Q41664760

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2006 nî lūn-bûn

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2006年論文

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2006年论文

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name

Factor VIII ectopically target ...... h-titer inhibitory antibodies.

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Factor VIII ectopically target ...... h-titer inhibitory antibodies.

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Factor VIII ectopically target ...... h-titer inhibitory antibodies.

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Journal of Clinical Investigation

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Factor VIII ectopically target ...... h-titer inhibitory antibodies.

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Brian C Cooley

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Clive W Wells

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David A Wilcox

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Drashti Desai

http://www.w3.org/2001/XMLSchema#string

Hartmut Weiler

http://www.w3.org/2001/XMLSchema#string

Jack Gorski

http://www.w3.org/2001/XMLSchema#string

Patricia A Morateck

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Qizhen Shi

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Robert R Montgomery

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Scot A Fahs

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P2860

Acquired anti-FVIII inhibitors in children

A mouse model of severe von Willebrand disease: defects in hemostasis and thrombosis

An enhanced method for post-embedding immunocytochemical staining which preserves cell membranes.

Suppression of erythro-megakaryocytopoiesis and the induction of reversible thrombocytopenia in mice transgenic for the thymidine kinase gene targeted by the platelet glycoprotein alpha IIb promoter.

A 2.7-kb portion of the 5' flanking region of the murine glycoprotein alphaIIb gene is transcriptionally active in primitive hematopoietic progenitor cells.

Advances toward gene therapy for hemophilia at the millennium.

Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy.

Cytokine-enhanced expression of glycoprotein Ib alpha in human endothelium.

Properties of anti-factor VIII inhibitor antibodies in hemophilia A patients.

Gene therapy: reality or myth for the global bleeding disorders community?

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1974-1982

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10.1172/JCI28416

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7

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116

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2006-07-01T00:00:00Z

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6962530

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16823491

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Coagulation factor VIII

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1483176

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