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Update on clinical gene therapy for hemophilia

Blood, 2019-01, Vol.133 (5), p.407-414

2019

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Immune Response Mechanisms against AAV Vectors in Animal Models

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.198-208

2020

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Clinical development of gene therapy: results and lessons from recent successes

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16034-16034, Article 16034

2016

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The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

Blood, 2011-06, Vol.117 (24), p.6459-6468

2011

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Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell–dependent induction of CD4+CD25+FoxP3+ Treg

Blood, 2015-05, Vol.125 (19), p.2937-2947

2015

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B Cell Depletion Eliminates FVIII Memory B Cells and Enhances AAV8-coF8 Immune Tolerance Induction When Combined With Rapamycin

Frontiers in immunology, 2020-06, Vol.11, p.1293-1293

2020

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Viral Vector Based Immunotherapy for Peanut Allergy

Viruses, 2024-07, Vol.16 (7), p.1125

2024

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Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy

PloS one, 2012-05, Vol.7 (5), p.e37671-e37671

2012

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Sweeten the deal: Glycopolymer-based engineering to modulate autoreactive T cell responses

Molecular therapy, 2023-11, Vol.31 (11), p.3119-3120

2023

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High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

Molecular therapy, 2010-12, Vol.18 (12), p.2048-2056

2010

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Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene Transfer

Journal of innate immunity, 2015-01, Vol.7 (3), p.302-314

2015

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Single-dose AAV vector gene immunotherapy to treat food allergy

Molecular therapy. Methods & clinical development, 2022-09, Vol.26, p.309-322

2022

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Complement System Response to Adeno-Associated Virus Vector Gene Therapy

Human gene therapy, 2024-07, Vol.35 (13-14), p.425-438

2024

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B cell focused transient immune suppression protocol for efficient AAV readministration to the liver

Molecular therapy. Methods & clinical development, 2024-03, Vol.32 (1), p.101216-101216, Article 101216

2024

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Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Journal of translational medicine, 2017-05, Vol.15 (1), p.94-94, Article 94

2017

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Development of Gene Transfer for Induction of Antigen-specific Tolerance

Molecular therapy. Methods & clinical development, 2014-04, Vol.1 (C), p.14013-14013, Article 14013

2014

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Potential for cellular stress response to hepatic factor VIII expression from AAV vector

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16063-16063, Article 16063

2016

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Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.98-107

2021

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Manipulating the microbiome to enhance oral tolerance in food allergy

Cellular immunology, 2022-12, Vol.382, p.104633-104633, Article 104633

2022

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Liver induced transgene tolerance with AAV vectors

Cellular immunology, 2019-08, Vol.342, p.103728-103728, Article 103728

2019

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Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity

PloS one, 2009-08, Vol.4 (8), p.e6376-e6376

2009

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Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia

Molecular therapy. Methods & clinical development, 2014-07, Vol.1 (C), p.14030, Article 14030

2014

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An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8 + T Cells

Molecular therapy. Methods & clinical development, 2017-06, Vol.5 (C), p.142-152

2017

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Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis

Molecular therapy, 2018-01, Vol.26 (1), p.173-183

2018

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Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells

Molecular therapy, 2020-03, Vol.28 (3), p.758-770

2020

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Update on clinical gene therapy for hemophilia

Immune Response Mechanisms against AAV Vectors in Animal Models

Clinical development of gene therapy: results and lessons from recent successes

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell–dependent induction of CD4+CD25+FoxP3+ Treg

B Cell Depletion Eliminates FVIII Memory B Cells and Enhances AAV8-coF8 Immune Tolerance Induction When Combined With Rapamycin

Viral Vector Based Immunotherapy for Peanut Allergy

Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy

Sweeten the deal: Glycopolymer-based engineering to modulate autoreactive T cell responses

High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene Transfer

Single-dose AAV vector gene immunotherapy to treat food allergy

Complement System Response to Adeno-Associated Virus Vector Gene Therapy

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver

Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Development of Gene Transfer for Induction of Antigen-specific Tolerance

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

Manipulating the microbiome to enhance oral tolerance in food allergy

Liver induced transgene tolerance with AAV vectors

Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity

Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia

An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8 + T Cells

Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis

Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells

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