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Higher Seroprevalence of Anti-Adeno-Associated Viral Vector Neutralizing Antibodies Among Racial Minorities in the United States

Human gene therapy, 2022-04, Vol.33 (7-8), p.442-450

2022

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Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia

Human gene therapy, 2013-01, Vol.24 (1), p.19-26

2013

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Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes

The Journal of immunology (1950), 2017-06, Vol.198 (12), p.4581-4587

2017

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Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies

Molecular therapy. Methods & clinical development, 2024-09, Vol.32 (3), p.101273, Article 101273

2024

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Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia

Molecular therapy. Methods & clinical development, 2021-09, Vol.22, p.1-10

2021

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Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials

Molecular therapy. Methods & clinical development, 2023-12, Vol.31, p.101119-101119, Article 101119

2023

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Inflammation Promotes the Loss of Adeno-Associated Virus–Mediated Transgene Expression in Mouse Liver

Gastroenterology (New York, N.Y. 1943), 2011-07, Vol.141 (1), p.348-357.e3

2011

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In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency

Molecular therapy. Methods & clinical development, 2022-03, Vol.24, p.367-378

2022

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A New Genetic Vaccine Platform Based on an Adeno-Associated Virus Isolated from a Rhesus Macaque

Journal of Virology, 2009-12, Vol.83 (24), p.12738-12750

2009

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Adeno-Associated Viruses Undergo Substantial Evolution in Primates during Natural Infections

Proceedings of the National Academy of Sciences - PNAS, 2003-05, Vol.100 (10), p.6081-6086

2003

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Host Immune Responses to Chronic Adenovirus Infections in Human and Nonhuman Primates

Journal of Virology, 2009-03, Vol.83 (6), p.2623-2631

2009

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Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial

Molecular therapy, 2020-03, Vol.28 (3), p.784-793

2020

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Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors

Journal of virology, 2018-10, Vol.92 (20)

2018

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Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver

Hepatology (Baltimore, Md.), 2009-08, Vol.50 (2), p.612-621

2009

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Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

Molecular therapy, 2023-03, Vol.31 (3), p.616-630

2023

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TLR9 signaling mediates adaptive immunity following systemic AAV gene therapy

Cellular immunology, 2019-12, Vol.346, p.103997-103997, Article 103997

2019

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Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

Molecular therapy, 2015-12, Vol.23 (12), p.1877-1887

2015

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Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes

Human gene therapy, 2023-06, Vol.34 (11-12), p.554-566

2023

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Global Seroprevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Serotypes of Relevance to Gene Therapy

Blood, 2022-11, Vol.140 (Supplement 1), p.10668-10670

2022

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Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

Proceedings of the National Academy of Sciences - PNAS, 2017-02, Vol.114 (7), p.1655-1659

2017

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Adeno-Associated Virus Mediated Delivery of a TFPI Antibody Restores Hemostasis in Mouse Model of Hemophilia

Blood, 2019-11, Vol.134 (Supplement_1), p.3357-3357

2019

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Biodistribution and Tolerability of AAV-PHP.B-CBh- SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

Human gene therapy, 2022-02, Vol.33 (3-4), p.175-187

2022

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Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

The Journal of clinical investigation, 2013-12, Vol.123 (12), p.5310-5318

2013

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AAV Vectors Expressing LDLR Gain-of-Function Variants Demonstrate Increased Efficacy in Mouse Models of Familial Hypercholesterolemia

Circulation research, 2014-08, Vol.115 (6), p.591-599

2014

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Alternative start sites downstream of nonsense mutations drive antigen presentation and tolerance induction to C-terminal epitopes

The Journal of immunology (1950), 2017-05, Vol.198 (12), p.4581-4587

2017

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Higher Seroprevalence of Anti-Adeno-Associated Viral Vector Neutralizing Antibodies Among Racial Minorities in the United States

Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia

Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes

Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies

Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia

Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials

Inflammation Promotes the Loss of Adeno-Associated Virus–Mediated Transgene Expression in Mouse Liver

In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency

A New Genetic Vaccine Platform Based on an Adeno-Associated Virus Isolated from a Rhesus Macaque

Adeno-Associated Viruses Undergo Substantial Evolution in Primates during Natural Infections

Host Immune Responses to Chronic Adenovirus Infections in Human and Nonhuman Primates

Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial

Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors

Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver

Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

TLR9 signaling mediates adaptive immunity following systemic AAV gene therapy

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes

Global Seroprevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) Serotypes of Relevance to Gene Therapy

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

Adeno-Associated Virus Mediated Delivery of a TFPI Antibody Restores Hemostasis in Mouse Model of Hemophilia

Biodistribution and Tolerability of AAV-PHP.B-CBh- SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

AAV Vectors Expressing LDLR Gain-of-Function Variants Demonstrate Increased Efficacy in Mouse Models of Familial Hypercholesterolemia

Alternative start sites downstream of nonsense mutations drive antigen presentation and tolerance induction to C-terminal epitopes

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