In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection
about
Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapyAlloantibodies to therapeutic factor VIII in hemophilia A: the role of von Willebrand factor in regulating factor VIII immunogenicityApoptotic effects of platelet factor VIII on megakaryopoiesis: implications for a modified human FVIII for platelet-based gene therapy.The immunogenicity of platelet-derived FVIII in hemophilia A mice with or without preexisting anti-FVIII immunityOverexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.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.Platelets are a safe way to deliver factor VIII. After 13 years of preclinical research it is now time for a clinical trial.Platelet-Targeted Gene Therapy for Hemophilia.Platelet Gene Therapy Promotes Targeted Peripheral Tolerance by Clonal Deletion and Induction of Antigen-Specific Regulatory T Cells
P2860
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P2860
In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection
description
2014 nî lūn-bûn
@nan
2014 թուականի Յուլիսին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի հուլիսին 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
In vivo enrichment of genetica ...... low multiplicity of infection
@ast
In vivo enrichment of genetica ...... low multiplicity of infection
@en
In vivo enrichment of genetica ...... low multiplicity of infection
@nl
type
label
In vivo enrichment of genetica ...... low multiplicity of infection
@ast
In vivo enrichment of genetica ...... low multiplicity of infection
@en
In vivo enrichment of genetica ...... low multiplicity of infection
@nl
prefLabel
In vivo enrichment of genetica ...... low multiplicity of infection
@ast
In vivo enrichment of genetica ...... low multiplicity of infection
@en
In vivo enrichment of genetica ...... low multiplicity of infection
@nl
P2093
P2860
P356
P1476
In vivo enrichment of genetica ...... low multiplicity of infection
@en
P2093
P2860
P304
P356
10.1111/JTH.12633
P577
2014-07-17T00:00:00Z