Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

Tetramer-Based Enrichment of Preexisting Anti-AAV8 CD8+ T Cells in Human Donors Allows the Detection of a TEMRA Subpopulation

(2020) Céline Vandamme et al.   Frontiers in Immunology

AAVcis-regulatory sequences are correlated with ocular toxicity

(2019) Wenjun Xiong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Impact of age and motor function in a phase 1/2A study of infants with SMA Type 1 receiving single-dose gene replacement therapy

(2019) Linda P. Lowes et al.   PEDIATRIC NEUROLOGY

Onasemnogene Abeparvovec: First Global Approval

(2019) Sheridan M. Hoy   DRUGS

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

Dynamics and genomic landscape of CD8+ T cells undergoing hepatic priming

(2019) Alexandre P. Bénéchet et al.   NATURE

O.40Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG)

(2019) R. Finkel et al.   NEUROMUSCULAR DISORDERS

Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design

(2019) Pierce J. Ogden et al.   SCIENCE

A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism

(2019) Marcus Davidsson et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients

(2018) Christian Leborgne et al.   CELLULAR IMMUNOLOGY

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Age-related seroprevalence of antibodies against AAV-LK03 in a UK population cohort

(2018) Dany Perocheau et al.   HUMAN GENE THERAPY

TLR2 Stimulation Strengthens Intrahepatic Myeloid-Derived Cell-Mediated T Cell Tolerance through Inducing Kupffer Cell Expansion and IL-10 Production

(2018) Jia Liu et al.   JOURNAL OF IMMUNOLOGY

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

(2018) Geoffrey D. Keeler et al.   MOLECULAR THERAPY

Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors

(2018) Roberto Calcedo et al.   Human Gene Therapy Methods

Pharmacology of Recombinant Adeno-associated Virus Production

(2018) Magalie Penaud-Budloo et al.   Molecular Therapy-Methods & Clinical Development

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

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

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

(2018) Klaudia Kuranda et al.   JOURNAL OF CLINICAL INVESTIGATION

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

(2018) Samiah Al‐Zaidy et al.   PEDIATRIC PULMONOLOGY

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

(2018) Amine Meliani et al.   Nature Communications

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

(2018) Jenny A. Greig et al.   Molecular Therapy-Methods & Clinical Development

Alloantigen gene transfer to hepatocytes promotes tolerance to pancreatic islet graft by inducing CD8 + regulatory T cells

(2017) Valentin Le Guen et al.   JOURNAL OF HEPATOLOGY

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

(2017) Marc Tardieu et al.   LANCET NEUROLOGY

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

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

(2017) Christian Mueller et al.   MOLECULAR THERAPY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2017) Roberto Calcedo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2017) Brett Palaschak et al.   Molecular Therapy-Methods & Clinical Development

Regulatory T cells and TLR9 activation shape antibody formation to a secreted transgene product in AAV muscle gene transfer

(2017) Roland W. Herzog et al.   CELLULAR IMMUNOLOGY

480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes

(2016) Jerry R. Mendell et al.   MOLECULAR THERAPY

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

(2015) M. Biswas et al.   BLOOD

The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer

(2015) Florence Boisgerault et al.   CURRENT GENE THERAPY

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

(2015) Paul E. Monahan et al.   HUMAN GENE THERAPY

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

(2015) Geoffrey L. Rogers et al.   Journal of Innate Immunity

Vaccinia virus as a subhelper for AAV replication and packaging

(2015) Andrea R Moore et al.   Molecular Therapy-Methods & Clinical Development

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

(2015) Daniel J Hui et al.   Molecular Therapy-Methods & Clinical Development

Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

(2014) Balaji Balakrishnan et al.   CURRENT GENE THERAPY

Hepatocytes induce Foxp3+ regulatory T cells by Notch signaling

(2014) Sven Burghardt et al.   JOURNAL OF LEUKOCYTE BIOLOGY

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Antigen expression level threshold tunes the fate of CD8 T cells during primary hepatic immune responses

(2014) S. S. Tay et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mapping the AAV Capsid Host Antibody Response toward the Development of Second Generation Gene Delivery Vectors

(2014) Yu-Shan Tseng et al.   Frontiers in Immunology

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

Hepatocytes Contribute to Immune Regulation in the Liver by Activation of the Notch Signaling Pathway in T Cells

(2013) S. Burghardt et al.   JOURNAL OF IMMUNOLOGY

C-Reactive Protein (CRP) Is Essential for Efficient Systemic Transduction of Recombinant Adeno-Associated Virus Vector 1 (rAAV-1) and rAAV-6 in Mice

(2013) J. Denard et al.   JOURNAL OF VIROLOGY

Manufacturing and Regulatory Strategies for Clinical AAV2-hRPE65

(2012) J. Fraser Wright et al.   CURRENT GENE THERAPY

Cytometry by Time-of-Flight Shows Combinatorial Cytokine Expression and Virus-Specific Cell Niches within a Continuum of CD8+ T Cell Phenotypes

(2012) Evan W. Newell et al.   IMMUNITY

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

rAAV-Mediated Tumorigenesis: Still Unresolved After an AAV Assault

(2012) Paul N Valdmanis et al.   MOLECULAR THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan 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

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

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

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

(2011) C Li et al.   GENE THERAPY

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

(2011) Marianna Hösel et al.   HEPATOLOGY

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

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

Hepatocyte entry leads to degradation of autoreactive CD8 T cells

(2011) V. Benseler et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Engineering of baculovirus vectors for the manufacture of virion-free biopharmaceuticals

(2010) Martin Marek et al.   BIOTECHNOLOGY AND BIOENGINEERING

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

Production, Purification and Characterization of Adeno-Associated Vectors

(2010) Eduard Ayuso et al.   CURRENT GENE THERAPY

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

(2010) Ekaterina Breous 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

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

(2009) E Ayuso et al.   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

Scalable Recombinant Adeno-Associated Virus Production Using Recombinant Herpes Simplex Virus Type 1 Coinfection of Suspension-Adapted Mammalian Cells

(2009) Darby L. Thomas 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

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

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

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

(2009) Lili Wang et al.   MOLECULAR THERAPY

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

Artificial Evolution with Adeno-Associated Viral Libraries

(2008) Hildegard Buning et al.   COMBINATORIAL CHEMISTRY & HIGH THROUGHPUT SCREENING

Intrahepatic Murine CD8 T-Cell Activation Associates With a Distinct Phenotype Leading to Bim-Dependent Death

(2008) Lauren E. Holz et al.   GASTROENTEROLOGY

Manufacturing and characterizing AAV-based vectors for use in clinical studies

(2008) J F Wright   GENE THERAPY

Adeno-associated virus integration: virus versus vector

(2008) R H Smith   GENE THERAPY

Mechanism of T cell tolerance induction by murine hepatic Kupffer cells

(2008) Qiang You et al.   HEPATOLOGY

Diverse IgG subclass responses to adeno-associated virus infection and vector administration

(2008) Samuel L. Murphy et al.   JOURNAL OF MEDICAL VIROLOGY

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

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

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz et al.   MOLECULAR THERAPY

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY