Challenges Posed by Immune Responses to AAV Vectors: Addressing Root Causes

Immune Responses to Viral Gene Therapy Vectors

(2020) Jamie L. Shirley et al.   MOLECULAR THERAPY

Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?

(2020) J. Fraser Wright   MOLECULAR THERAPY

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

(2020) Berangere Bertin et al.   Scientific Reports

Revisiting the “New” Inflammatory Toxicities of Adeno-Associated Virus Vectors

(2020) Terence R. Flotte   HUMAN GENE THERAPY

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

(2020) Christian Leborgne et al.   NATURE MEDICINE

Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future

(2020) Agnieszka Łoboda et al.   Pharmacological Reports

Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates

(2020) Benjamin J. Samelson-Jones et al.   Molecular Therapy-Methods & Clinical Development

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression

(2020) Barbara A Konkle et al.   BLOOD

Methylation Status of the Adeno-Associated Virus Type 2 (AAV2)

(2019) Renáta Tóth et al.   Viruses-Basel

Adeno-associated Virus (AAV) versus Immune Response

(2019) Joseph Rabinowitz et al.   Viruses-Basel

Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors

(2019) Anita Kruzik et al.   Molecular Therapy-Methods & Clinical Development

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

(2019) Jamie L. Shirley et al.   MOLECULAR THERAPY

The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer

(2019) ZhiQuan Xiang et al.   MOLECULAR THERAPY

Effects of codon optimization on coagulation factor IX translation and structure: Implications for protein and gene therapies

(2019) Aikaterini Alexaki et al.   Scientific Reports

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

(2019) Natalie F. Nidetz et al.   PHARMACOLOGY & THERAPEUTICS

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle

(2019) Jérôme Poupiot et al.   Molecular Therapy-Methods & Clinical Development

Toll-like Receptor 9 Contains Two DNA Binding Sites that Function Cooperatively to Promote Receptor Dimerization and Activation

(2018) Umeharu Ohto et al.   IMMUNITY

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

(2018) Zachary Fitzpatrick et al.   Molecular Therapy-Methods & Clinical Development

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

(2018) Amine Meliani et al.   Nature Communications

The Balance between CD8 + T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose

(2017) Sandeep R.P. Kumar et al.   MOLECULAR THERAPY

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy

(2017) Haiyan Fu et al.   Human Gene Therapy Clinical Development

Structural basis of CpG and inhibitory DNA recognition by Toll-like receptor 9

(2015) Umeharu Ohto et al.   NATURE

Complement and Its Receptors: New Insights into Human Disease

(2014) V. Michael Holers   Annual Review of Immunology

A critical analysis of codon optimization in human therapeutics

(2014) Vincent P. Mauro et al.   TRENDS IN MOLECULAR MEDICINE

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

(2014) M Corti et al.   Molecular Therapy-Methods & Clinical Development

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

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

CD8+ T Cell Recognition of Epitopes Within the Capsid of Adeno-associated Virus 8–based Gene Transfer Vectors Depends on Vectors’ Genome

(2013) Te-Lang Wu et al.   MOLECULAR THERAPY

Humoral Immune Response to AAV

(2013) Roberto Calcedo et al.   Frontiers in Immunology

CpG DNA as a vaccine adjuvant

(2011) Christian Bode et al.   Expert Review of Vaccines

Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors

(2011) Marianna Hösel et al.   HEPATOLOGY

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet 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

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

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

Adeno-Associated Viral Vector-Mediated Transgene Expression Is Independent of DNA Methylation in Primate Liver and Skeletal Muscle

(2011) Adrien Léger et al.   PLoS One

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

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

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

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

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

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

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

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