Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.
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Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia BAnimal models of hemophiliaGene therapy for hemophiliaImmune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.Immune tolerance induction to factor IX through B cell gene transfer: TLR9 signaling delineates between tolerogenic and immunogenic B cells.GITR engagement preferentially enhances proliferation of functionally competent CD4+CD25+FoxP3+ regulatory T cellsAnti-inflammatory loaded poly-lactic glycolic acid nanoparticle formulations to enhance myocardial gene transfer: an in-vitro assessment of a drug/gene combination therapeutic approach for direct injection.Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice.Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.Suppression of inhibitor formation against FVIII in a murine model of hemophilia A by oral delivery of antigens bioencapsulated in plant cellsTransient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy.Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them.High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines.Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia.Portal vein delivery of viral vectors for gene therapy for hemophilia.Pompe disease gene therapy.Immune responses to human factor IX in haemophilia B mice of different genetic backgrounds are distinct and modified by TLR4.The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver.Plant-based oral tolerance to hemophilia therapy employs a complex immune regulatory response including LAP+CD4+ T cellsIntrinsic transgene immunogenicity gears CD8(+) T-cell priming after rAAV-mediated muscle gene transfer.Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene TransferGene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation.Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.Host Anti-antibody Responses Following Adeno-associated Virus-mediated Delivery of Antibodies Against HIV and SIV in Rhesus MonkeysOral delivery of human biopharmaceuticals, autoantigens and vaccine antigens bioencapsulated in plant cells.Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice.Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies.Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodiesGene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.The complex and evolving story of T cell activation to AAV vector-encoded transgene products.Innate Immune Responses to AAV VectorsDevelopment of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia.The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer.State of play and clinical prospects of antibody gene transfer.Complexity of immune responses to AAV transgene products - Example of factor IX.Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia.Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes.Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells.Dynamics of antigen presentation to transgene product-specific CD4+ T cells and of Treg induction upon hepatic AAV gene transfer.
P2860
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P2860
Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh
2009年學術文章
@zh-hant
name
Impact of the underlying mutat ...... gene therapy for hemophilia B.
@en
Impact of the underlying mutat ...... gene therapy for hemophilia B.
@nl
type
label
Impact of the underlying mutat ...... gene therapy for hemophilia B.
@en
Impact of the underlying mutat ...... gene therapy for hemophilia B.
@nl
prefLabel
Impact of the underlying mutat ...... gene therapy for hemophilia B.
@en
Impact of the underlying mutat ...... gene therapy for hemophilia B.
@nl
P2093
P2860
P50
P921
P356
P1433
P1476
Impact of the underlying mutat ...... gene therapy for hemophilia B
@en
P2093
Babak Moghimi
Hildegund C J Ertl
Mario Cooper
Roland W Herzog
Shangzhen Zhou
P2860
P304
P356
10.1038/MT.2009.159
P577
2009-07-14T00:00:00Z