Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper

Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?

(2022) Takashi Kei Kishimoto et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Directed evolution of adeno-associated virus 5 capsid enables specific liver tropism

(2022) Yuqiu Wang et al.   Molecular Therapy-Nucleic Acids

Differential T cell immune responses to deamidated adeno-associated virus vector

(2022) So Jin Bing et al.   Molecular Therapy-Methods & Clinical Development

Confirmatory detection of neutralizing antibodies to AAV gene therapy using a cell-based transduction inhibition assay

(2022) Theresa Kasprzyk et al.   Molecular Therapy-Methods & Clinical Development

Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates

(2021) Fan Yang et al.   GENE THERAPY

Overcoming innate immune barriers that impede AAV gene therapy vectors

(2021) Manish Muhuri et al.   JOURNAL OF CLINICAL INVESTIGATION

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

(2021) Ying Kai Chan et al.   Science Translational Medicine

Sutimlimab in Cold Agglutinin Disease

(2021) Alexander Röth et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2021) Bradley A. Hamilton et al.   Frontiers in Immunology

T Cell-Mediated Immune Responses to AAV and AAV Vectors

(2021) Hildegund C. J. Ertl   Frontiers in Immunology

Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions

(2021) Thomas Weber   Frontiers in Immunology

Immunomodulation in Administration of rAAV: Preclinical and Clinical Adjuvant Pharmacotherapies

(2021) Wing Sum Chu et al.   Frontiers in Immunology

Characterization of Recombinant Adeno-Associated Viruses (rAAVs) for Gene Therapy Using Orthogonal Techniques

(2021) Liam Cole et al.   Pharmaceutics

Evaluation of the Humoral Response to Adeno-Associated Virus-Based Gene Therapy Modalities Using Total Antibody Assays

(2021) Boris Gorovits et al.   AAPS Journal

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

(2021) Mohammadsharif Tabebordbar et al.   CELL

AAV integration in human hepatocytes

(2021) Dhwanil A. Dalwadi et al.   MOLECULAR THERAPY

Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method

(2021) Kathryn S. Patton et al.   Molecular Therapy-Methods & Clinical Development

A sensitive and reproducible cell-based assay via secNanoLuc to detect neutralizing antibody against adeno-associated virus vector capsid

(2021) Nemekhbayar Baatartsogt et al.   Molecular Therapy-Methods & Clinical Development

Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain

(2021) Megan S. Keiser et al.   NATURE MEDICINE

Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays

(2020) Boris Gorovits et al.   AAPS Journal

Blockade of the costimulatory CD28‐B7 family signal axis enables repeated application of AAV8 gene vectors

(2020) Marco Frentsch et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

(2020) Gwladys Gernoux et al.   MOLECULAR THERAPY

Immune Responses to Viral Gene Therapy Vectors

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

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

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

(2020) J. Fraser Wright   MOLECULAR THERAPY

Mechanisms of action of hydroxychloroquine and chloroquine: implications for rheumatology

(2020) Eva Schrezenmeier et al.   Nature Reviews Rheumatology

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

Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates

(2020) Glenn Yiu et al.   Molecular Therapy-Methods & Clinical Development

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

(2020) Christian Leborgne et al.   NATURE MEDICINE

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

(2020) Giuseppe Ronzitti et al.   Frontiers in Immunology

Letter to the Editor

(2020) Perry B. Shieh et al.   HUMAN GENE THERAPY

Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll

(2020) J. Fraser Wright   MOLECULAR THERAPY

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

(2020) Christian Mueller et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2020) Agnieszka Łoboda et al.   Pharmacological Reports

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

(2020) Marti Cabanes-Creus 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

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

(2020) Juliette Hordeaux et al.   HUMAN GENE THERAPY

Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme

(2020) Zachary C. Elmore et al.   JCI Insight

Quality Control Testing, Characterization and Critical Quality Attributes of Adeno‐Associated Virus Vectors Used for Human Gene Therapy

(2020) J. Fraser Wright   Biotechnology Journal

Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

(2020) Brian R. Long et al.   MOLECULAR THERAPY

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

(2020) Deepa Chand et al.   JOURNAL OF HEPATOLOGY

Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

(2020) Deepa H. Chand et al.   JOURNAL OF PEDIATRICS

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors

(2019) Hildegard Büning et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated Virus (AAV) versus Immune Response

(2019) Joseph Rabinowitz et al.   Viruses-Basel

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Ravulizumab: First Global Approval

(2019) Kate McKeage   DRUGS

Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys

(2019) Juliette Hordeaux et al.   HUMAN GENE THERAPY

Next-generation AAV vectors - don't judge a virus (only) by its cover

(2019) Claire Domenger et al.   HUMAN MOLECULAR GENETICS

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

(2019) Brian R. Long et al.   Molecular Therapy-Methods & Clinical Development

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

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

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

(2019) Laurel C. Chandler et al.   Molecular Therapy-Methods & Clinical Development

Compendium of current complement therapeutics

(2019) Wioleta M Zelek et al.   MOLECULAR IMMUNOLOGY

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

TLR9 signaling mediates adaptive immunity following systemic AAV gene therapy

(2019) Scott N. Ashley et al.   CELLULAR IMMUNOLOGY

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

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

Immune Response Mechanisms against AAV Vectors in Animal Models

(2019) Ashley T. Martino et al.   Molecular Therapy-Methods & Clinical Development

IdeS (Imlifidase)

(2018) Bonnie E. Lonze et al.   ANNALS OF SURGERY

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

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

Impact of neutralizing antibodies against AAV is a key consideration in gene transfer to nonhuman primates

(2018) Weidong Xiao et al.   NATURE MEDICINE

Immunogenicity testing of therapeutic antibodies in ocular fluids after intravitreal injection

(2018) Uwe Wessels et al.   Bioanalysis

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

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

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

Dosage thresholds and influence of transgene cassette in AAV-related toxicity

(2018) Hanen Khabou et al.   HUMAN GENE THERAPY

Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study

(2018) Jong Wook Lee et al.   BLOOD

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

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

Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

(2018) Basel T. Assaf et al.   TOXICOLOGIC PATHOLOGY

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

(2018) Dan Wang et al.   Molecular Therapy-Methods & Clinical Development

Rapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay

(2018) Ping Guo et al.   Molecular Therapy-Methods & Clinical Development

Strategy to detect pre-existing immunity to AAV gene therapy

(2017) L Falese et al.   GENE THERAPY

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial

(2017) Céline Vandamme et al.   HUMAN GENE THERAPY

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

(2017) Hildegund C.J. Ertl et al.   HUMAN GENE THERAPY

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

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

AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye

(2017) Felix F. Reichel 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

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

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

(2017) Manuela Corti et al.   Human Gene Therapy Clinical Development

Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting

(2017) Victoria M. Velazquez et al.   Molecular Therapy-Methods & Clinical Development

Preclinical models to assess the immunogenicity of AAV vectors

(2017) Hildegund C.J. Ertl   CELLULAR IMMUNOLOGY

Complexity of immune responses to AAV transgene products – Example of factor IX

(2017) Roland W. Herzog   CELLULAR IMMUNOLOGY

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

Pre-existing Antibody: Biotherapeutic Modality-Based Review

(2016) Boris Gorovits et al.   AAPS Journal

Strategies to Determine Assay Format for the Assessment of Neutralizing Antibody Responses to Biotherapeutics

(2016) Bonnie Wu et al.   AAPS Journal

Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis

(2016) Daniel Gaudet et al.   HUMAN GENE THERAPY

High Prevalence of Infectious Adeno-associated Virus (AAV) in Human Peripheral Blood Mononuclear Cells Indicative of T Lymphocytes as Sites of AAV Persistence

(2016) Daniela Hüser et al.   JOURNAL OF VIROLOGY

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

CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy

(2015) Kathryn L. Berrier et al.   GENETICS IN MEDICINE

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

Preexisting Neutralizing Antibodies to Adeno-Associated Virus Capsids in Large Animals Other Than Monkeys May ConfoundIn VivoGene Therapy Studies

(2015) Roberto Calcedo et al.   Human Gene Therapy Methods

Determination of Anti-Adeno-Associated Virus Vector Neutralizing Antibody Titer with anIn VitroReporter System

(2015) Amine Meliani et al.   Human Gene Therapy Methods

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

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

Complement and Its Receptors: New Insights into Human Disease

(2014) V. Michael Holers   Annual Review of Immunology

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

(2014) Balaji Balakrishnan et al.   CURRENT GENE THERAPY

Vector Design Tour de Force: Integrating Combinatorial and Rational Approaches to Derive Novel Adeno-associated Virus Variants

(2014) Damien Marsic et al.   MOLECULAR THERAPY

AAV Empty Capsids: For Better or for Worse?

(2014) J Fraser Wright   MOLECULAR THERAPY

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

mTOR kinase inhibitors promote antibody class switching via mTORC2 inhibition

(2014) Jose J. Limon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Clinical evaluation of CpG oligonucleotides as adjuvants for vaccines targeting infectious diseases and cancer

(2014) Julia Scheiermann et al.   VACCINE

IL-6 in Inflammation, Immunity, and Disease

(2014) T. Tanaka et al.   Cold Spring Harbor Perspectives in Biology

Pre-Clinical Assessment of Immune Responses to Adeno-Associated Virus (AAV) Vectors

(2014) Etiena Basner-Tschakarjan et al.   Frontiers in Immunology

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside

(2014) Valerie Ferreira et al.   Frontiers in Immunology

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

(2013) A. T. Martino et al.   BLOOD

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

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

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

(2013) Chengwen Li 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

Mechanistic Insights into the Enhancement of Adeno-Associated Virus Transduction by Proteasome Inhibitors

(2013) Angela M. Mitchell et al.   JOURNAL OF VIROLOGY

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

Gene therapy – Why can it fail?

(2013) Georg F. Weber   MEDICAL HYPOTHESES

Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery

(2013) LG Chicoine et al.   MOLECULAR THERAPY

Terminal Complement Inhibitor Eculizumab in Atypical Hemolytic–Uremic Syndrome

(2013) C.M. Legendre et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

(2013) Vedell Louis Jeune et al.   Human Gene Therapy Methods

Differing Effects of Rapamycin or Calcineurin Inhibitor on T-Regulatory Cells in Pediatric Liver and Kidney Transplant Recipients

(2012) T. Akimova et al.   AMERICAN JOURNAL OF TRANSPLANTATION

Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction

(2012) A L H Arnett et al.   GENE THERAPY

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Human Galectin 3 Binding Protein Interacts with Recombinant Adeno-Associated Virus Type 6

(2012) J. Denard et al.   JOURNAL OF VIROLOGY

In Vitro Effects of Cyclosporine A and Tacrolimus on Regulatory T-Cell Proliferation and Function

(2012) Céline Miroux et al.   TRANSPLANTATION

ELISPOT Assay for Monitoring Cytotoxic T Lymphocytes (CTL) Activity in Cancer Vaccine Clinical Trials

(2012) Anatoli M. Malyguine et al.   Cells

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

ELISpot for measuring human immune responses to vaccines

(2011) Meredith Slota et al.   Expert Review of Vaccines

CpG DNA as a vaccine adjuvant

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

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

Mycophenolate Mofetil Impairs Transduction of Single-Stranded Adeno-Associated Viral Vectors

(2011) Paula S. Montenegro-Miranda et al.   HUMAN 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

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

Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease

(2011) Márcia Séllos-Moura et al.   JOURNAL OF IMMUNOLOGICAL METHODS

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

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

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

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

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

(2010) Paul E Monahan 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

The mTOR Kinase Differentially Regulates Effector and Regulatory T Cell Lineage Commitment

(2009) Greg M. Delgoffe et al.   IMMUNITY

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

ELISPOT assays provide reproducible results among different laboratories for T-cell immune monitoring—even in hands of ELISPOT-inexperienced investigators

(2009) W. Zhang et al.   Journal of Immunotoxicology

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

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

Comparison of Neutralizing Antibody Assays for Receptor Binding and Enzyme Activity of the Enzyme Replacement Therapeutic Naglazyme® (Galsulfase)

(2008) Joleen T. White et al.   AAPS Journal

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

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

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