Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

Global seroprevalence of pre-existing immunity against AAV5 and other AAV serotypes in people with hemophilia A

(2022) Robert Klamroth et al.   HUMAN GENE THERAPY

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

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

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

(2022) Jerry R. Mendell et al.   Molecular Therapy-Methods & Clinical Development

High concordance of ELISA and neutralization assays allows for the detection of antibodies to individual AAV serotypes

(2022) Matthew R. Gardner et al.   Molecular Therapy-Methods & Clinical Development

Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials

(2022) Liching Cao et al.   GENE THERAPY

IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice

(2022) Tierra A. Bobo et al.   GENE THERAPY

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

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

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

(2021) Thomas Weber   Frontiers in Immunology

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

(2021) Boris Gorovits et al.   AAPS Journal

Gene therapies and COVID-19 vaccines: a necessary discussion in relation with viral vector-based approaches

(2021) Angel Aledo-Serrano et al.   Orphanet Journal of Rare Diseases

Higher Seroprevalence of Anti-Adeno-Associated Viral Vector Neutralizing Antibodies Among Racial Minorities in the United States

(2021) Arpana Khatri et al.   HUMAN GENE THERAPY

Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

(2021) Thomas Leth Jensen et al.   Frontiers in Molecular Neuroscience

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

(2020) Boris Gorovits et al.   AAPS Journal

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

(2020) Christian Leborgne et al.   NATURE MEDICINE

Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay

(2020) Karina Krotova et al.   HUMAN GENE THERAPY

Changes in Human Milk Immunoglobulin Profile During Prolonged Lactation

(2020) Matylda Czosnykowska-Łukacka et al.   Frontiers in Pediatrics

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

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

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

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

B cell depletion therapies in autoimmune disease: advances and mechanistic insights

(2020) Dennis S. W. Lee et al.   NATURE REVIEWS DRUG DISCOVERY

Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity

(2020) Moanaro Biswas et al.   Molecular Therapy-Methods & Clinical Development

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

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

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

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

Therapeutic hFIX activity achieved after single AAV5-hFIX treatment in hemophilia B patients and NHPs with pre-existing anti-AAV5 NABs

(2019) Anna Majowicz et al.   Molecular Therapy-Methods & Clinical Development

The Neonatal Fc Receptor (FcRn): A Misnomer?

(2019) Michal Pyzik et al.   Frontiers in Immunology

Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation

(2019) Aaron S. Meadows 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

Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia

(2019) Kavitha Rajavel et al.   BLOOD

Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia

(2019) Adrian C. Newland et al.   AMERICAN JOURNAL OF HEMATOLOGY

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

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

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

(2018) Christian Leborgne et al.   CELLULAR IMMUNOLOGY

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

(2018) Dany Perocheau et al.   HUMAN GENE THERAPY

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

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

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

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

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

(2017) Julien Baruteau et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Adeno-associated Virus (AAV) Serotypes Have Distinctive Interactions with Domains of the Cellular AAV Receptor

(2017) Sirika Pillay et al.   JOURNAL OF VIROLOGY

IgG Endopeptidase in Highly Sensitized Patients Undergoing Transplantation

(2017) Stanley C. Jordan 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

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

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

30. Modulation of Intracellular Calcium Enhances AAV Transduction in the CNS

(2016) Garrett Berry et al.   MOLECULAR THERAPY

Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques

(2016) Jenny A. Greig et al.   VACCINE

In vivo tissue-tropism of adeno-associated viral vectors

(2016) Arun Srivastava   Current Opinion in Virology

Effect of Type 2 Diabetes Mellitus and Diabetic Nephropathy on IgG Pharmacokinetics and Subcutaneous Bioavailability in the Rat

(2015) Gurkishan S. Chadha et al.   AAPS Journal

Strategies to circumvent humoral immunity to adeno-associated viral vectors

(2015) Longping V Tse et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Prediction of adeno-associated virus neutralizing antibody activity for clinical application

(2015) M Wang et al.   GENE THERAPY

Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure

(2015) B Greenberg et al.   GENE THERAPY

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

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

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

(2014) R. Jude Samulski et al.   Annual Review of Virology

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

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals

(2013) Jun Mimuro et al.   JOURNAL OF MEDICAL VIROLOGY

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

(2013) LG Chicoine et al.   MOLECULAR THERAPY

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Overview of Complement Activation and Regulation

(2013) Marina Noris et al.   SEMINARS IN NEPHROLOGY

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

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

Humoral Immune Response to AAV

(2013) Roberto Calcedo et al.   Frontiers in Immunology

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

(2012) F Mingozzi et al.   GENE THERAPY

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

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

(2012) Federico Mingozzi et al.   MOLECULAR THERAPY

Minimizing the Inhibitory Effect of Neutralizing Antibody for Efficient Gene Expression in the Liver With Adeno-associated Virus 8 Vectors

(2012) Jun Mimuro et al.   MOLECULAR THERAPY

IgG Placental Transfer in Healthy and Pathological Pregnancies

(2011) Patricia Palmeira et al.   Clinical & Developmental Immunology

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

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

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

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

Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

(2010) Gregory D Hurlbut et al.   MOLECULAR THERAPY

Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness

(2010) D. Amado et al.   Science Translational Medicine

A single course of rituximab does not abrogate anti-infliximab antibodies in patients with rheumatoid arthritis

(2009) B J F van den Bemt et al.   ANNALS OF THE RHEUMATIC DISEASES

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

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

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

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

(2009) Lili Wang et al.   MOLECULAR THERAPY

Adeno-associated virus integration: virus versus vector

(2008) R H Smith   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

Recombinant Adeno-associated Virus Transduction and Integration

(2008) Brian R Schultz et al.   MOLECULAR THERAPY