Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

(2013) A. T. Martino et al.   BLOOD

Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

(2013) Chengwen Li et al.   JOURNAL OF CLINICAL INVESTIGATION

Activation of the Cellular Unfolded Protein Response by Recombinant Adeno-Associated Virus Vectors

(2013) Balaji Balakrishnan et al.   PLoS One

Targeted Modifications in Adeno-Associated Virus Serotype 8 Capsid Improves Its Hepatic Gene Transfer Efficiency In Vivo

(2013) Dwaipayan Sen et al.   Human Gene Therapy Methods

Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo

(2013) Nishanth Gabriel et al.   Human Gene Therapy Methods

Intracellular transport of recombinant adeno-associated virus vectors

(2012) M Nonnenmacher et al.   GENE THERAPY

Assessment of a passive immunity mouse model to quantitatively analyze the impact of neutralizing antibodies on adeno-associated virus-mediated gene transfer

(2012) Lan Sun et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Humoral and Cellular Capsid-Specific Immune Responses to Adeno-Associated Virus Type 1 in Randomized Healthy Donors

(2012) P. Veron et al.   JOURNAL OF IMMUNOLOGY

Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

(2012) C. Li et al.   JOURNAL OF VIROLOGY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

(2012) Brandon K. Sack et al.   PLoS One

Proteasome Inhibition Is Partially Effective in Attenuating Pre-Existing Immunity against Recombinant Adeno-Associated Viral Vectors

(2012) Jozsef Karman et al.   PLoS One

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

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

Current Advances in Retroviral Gene Therapy

(2011) Youngsuk Yi et al.   CURRENT GENE THERAPY

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

(2011) J H McIntosh et al.   GENE THERAPY

Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors

(2011) Lili Wang et al.   HUMAN GENE THERAPY

Examining the cross-reactivity and neutralization mechanisms of a panel of mAbs against adeno-associated virus serotypes 1 and 5

(2011) C. E. Harbison et al.   JOURNAL OF GENERAL VIROLOGY

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

(2011) Peter A LeWitt et al.   LANCET NEUROLOGY

Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates

(2011) Hua Li et al.   MOLECULAR THERAPY

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

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

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

The Unfolded Protein Response: How Protein Folding Became a Restrictive Aspect for Innate Immunity and B Lymphocytes1

(2011) C. Z. F. Costa et al.   SCANDINAVIAN JOURNAL OF IMMUNOLOGY

Improved Immunological Tolerance Following Combination Therapy with CTLA-4/Ig and AAV-Mediated PD-L1/2 Muscle Gene Transfer

(2011) Sahil Adriouch et al.   Frontiers in Microbiology

Enhancing the clinical potential of AAV vectors by capsid engineering to evade pre-existing immunity

(2011) Melissa Bartel   Frontiers in Microbiology

Innate Immune Responses to AAV Vectors

(2011) Geoffrey L. Rogers et al.   Frontiers in Microbiology

Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver

(2010) C Qiao et al.   GENE THERAPY

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

BALB/c Mice Show Impaired Hepatic Tolerogenic Response Following AAV Gene Transfer to the Liver

(2010) Ekaterina Breous 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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

Pathogen Recognition and Inflammatory Signaling in Innate Immune Defenses

(2009) T. H. Mogensen   CLINICAL MICROBIOLOGY REVIEWS

MHC molecules and microbial antigen processing in phagosomes

(2009) Lakshmi Ramachandra et al.   CURRENT OPINION IN IMMUNOLOGY

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

Transient Transfection Methods for Clinical Adeno-Associated Viral Vector Production

(2009) J. Fraser Wright   HUMAN GENE THERAPY

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION

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

Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8 + T Cell Response to Vector Encoded Proteins

(2009) Lauren E. Mays et al.   JOURNAL OF IMMUNOLOGY

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Induction of Immune Tolerance to FIX Following Muscular AAV Gene Transfer Is AAV-dose/FIX-level Dependent

(2009) Meagan E Kelly et al.   MOLECULAR THERAPY

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

Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery

(2009) James T Koerber et al.   MOLECULAR THERAPY

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

Cellular immune response to cryptic epitopes during therapeutic gene transfer

(2009) C. Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Optimization of stealth adeno-associated virus vectors by randomization of immunogenic epitopes

(2009) Stephan Maersch et al.   VIROLOGY

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

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Applications of Lentiviral Vectors for Biology and Gene Therapy of Neurological Disorders

(2008) Cecilia Lundberg et al.   CURRENT GENE THERAPY

Immunity to adeno-associated virus vectors in animals and humans: a continued challenge

(2008) A K Zaiss et al.   GENE THERAPY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Complement Is an Essential Component of the Immune Response to Adeno-Associated Virus Vectors

(2008) A. K. Zaiss et al.   JOURNAL OF VIROLOGY

Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles

(2008) Stéphanie Lorain et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Spatial and mechanistic separation of cross-presentation and endogenous antigen presentation

(2008) Sven Burgdorf et al.   NATURE IMMUNOLOGY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA