Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B

Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

(2013) Andrea Annoni et al.   EMBO Molecular Medicine

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

(2013) Christian Mueller et al.   JOURNAL OF CLINICAL INVESTIGATION

CpG-depleted adeno-associated virus vectors evade immune detection

(2013) Susan M. Faust et al.   JOURNAL OF CLINICAL INVESTIGATION

Differential Type I Interferon-dependent Transgene Silencing of Helper-dependent Adenoviral vs. Adeno-associated Viral Vectors In Vivo

(2013) Masataka Suzuki et al.   MOLECULAR THERAPY

IL12-Mediated Liver Inflammation Reduces the Formation of AAV Transcriptionally Active Forms but Has No Effect over Preexisting AAV Transgene Expression

(2013) Irene Gil-Fariña et al.   PLoS One

The gene therapy journey for hemophilia: are we there yet?

(2012) K. A. High   BLOOD

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

MyD88 Signaling in B Cells Regulates the Production of Th1-dependent Antibodies to AAV

(2012) Muriel Sudres et al.   MOLECULAR THERAPY

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

(2011) A. T. Martino et al.   BLOOD

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

(2011) Janka Mátrai et al.   HEPATOLOGY

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

Toll-like Receptors and Their Crosstalk with Other Innate Receptors in Infection and Immunity

(2011) Taro Kawai et al.   IMMUNITY

Nonredundant Roles of IL-10 and TGF-β in Suppression of Immune Responses to Hepatic AAV-Factor IX Gene Transfer

(2011) Brad E Hoffman et al.   MOLECULAR THERAPY

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Self-complementary AAVs Induce More Potent Transgene Product-specific Immune Responses Compared to a Single-stranded Genome

(2011) TeLang Wu et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Activation of the NF-κB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy

(2011) Giridhara R. Jayandharan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges

(2011) Deepak Raj et al.   Expert Review of Hematology

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

(2010) V. R. Arruda et al.   BLOOD

GITR engagement preferentially enhances proliferation of functionally competent CD4+CD25+FoxP3+ regulatory T cells

(2010) Gongxian Liao et al.   INTERNATIONAL IMMUNOLOGY

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

(2010) David M Markusic et al.   MOLECULAR THERAPY

AAV Vectors Avoid Inflammatory Signals Necessary to Render Transduced Hepatocyte Targets for Destructive T Cells

(2010) Suryanarayan Somanathan et al.   MOLECULAR THERAPY

Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice

(2010) D. Verma et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hepatic Gene Transfer as a Means of Tolerance Induction to Transgene Products

(2009) Paul LoDuca et al.   CURRENT GENE THERAPY

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

(2009) Ekaterina Breous et al.   HEPATOLOGY

Improved Induction of Immune Tolerance to Factor IX by Hepatic AAV-8 Gene Transfer

(2009) Mario Cooper et al.   HUMAN GENE THERAPY

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

(2009) Jiangao Zhu et al.   JOURNAL OF CLINICAL INVESTIGATION

Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells

(2009) S. NAYAK et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Impact of the Underlying Mutation and the Route of Vector Administration on Immune Responses to Factor IX in Gene Therapy for Hemophilia B

(2009) Ou Cao et al.   MOLECULAR THERAPY

Tolerance Induction to Cytoplasmic β-Galactosidase by Hepatic AAV Gene Transfer — Implications for Antigen Presentation and Immunotoxicity

(2009) Ashley T. Martino et al.   PLoS One

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

(2008) G. P. Niemeyer et al.   BLOOD

Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy

(2008) V. M. Velazquez et al.   BLOOD

Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

(2007) Zhijian Wu et al.   MOLECULAR THERAPY