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
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Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B miceRecent advancements in erythrocytes, platelets, and albumin as delivery systemsIn utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical applicationMegakaryocyte- and megakaryocyte precursor-related gene therapiesHemophilia A: an ideal disease to correct in uteroAnimal models of hemophiliaHemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectorsCorrection of murine ADAMTS13 deficiency by hematopoietic progenitor cell-mediated gene therapyClinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A.In vivo efficacy of platelet-delivered, high specific activity factor VIII variantsA conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infectionFactor 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 treatmentsDevelopment of Gene Transfer for Induction of Antigen-specific Tolerance.Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.Profile of efraloctocog alfa and its potential in the treatment of 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.Role of bone marrow transplantation for correcting hemophilia A in mice.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 DeliveryThe immunogenicity of platelet-derived FVIII in hemophilia A mice with or without preexisting anti-FVIII immunityFactor VIII inhibitors: von Willebrand factor makes a difference in vitro and in vivoEctopic platelet-delivered factor (F) VIII for the treatment of Hemophilia A: Plasma and platelet FVIII, is it all the same?Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells.Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome.Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina.Gene therapy for haemophilia: a long and winding road.Platelet and endothelial expression of clotting factors for the treatment of hemophilia.Current status of haemophilia gene therapy.Novel investigations on the protective role of the FVIII/VWF complex in inhibitor development.Alternative strategies for gene therapy of hemophilia.Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia.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.Immune tolerance induced by platelet-targeted factor VIII gene therapy in hemophilia A mice is CD4 T cell mediated.
P2860
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P2860
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
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
2008年论文
@zh
2008年论文
@zh-cn
name
Syngeneic transplantation of h ...... ith preexisting FVIII immunity
@en
type
label
Syngeneic transplantation of h ...... ith preexisting FVIII immunity
@en
prefLabel
Syngeneic transplantation of h ...... ith preexisting FVIII immunity
@en
P2093
P2860
P921
P1433
P1476
Syngeneic transplantation of h ...... ith preexisting FVIII immunity
@en
P2093
David A Wilcox
Erin L Kuether
Hartmut Weiler
Nicole Mareno
Patricia A Morateck
Qizhen Shi
Robert R Montgomery
Scot A Fahs
P2860
P304
P356
10.1182/BLOOD-2008-02-138214
P407
P577
2008-05-21T00:00:00Z