Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques

Etranacogene Dezaparvovec: First Approval

(2023) Young-A Heo   DRUGS

P17 Safety and efficacy of pre-treatment with imlifidase prior to AAV-based gene therapy in non-human primates with pre-existing anti-AAVrh74 antibodies

(2023) R. Potter et al.   NEUROMUSCULAR DISORDERS

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

Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression

(2022) Lili Wang et al.   Molecular Therapy-Methods & Clinical Development

Valoctocogene Roxaparvovec: First Approval

(2022) Hannah A. Blair   DRUGS

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

(2021) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2020) Boris Gorovits et al.   AAPS Journal

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

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

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

(2020) Berangere Bertin et al.   Scientific Reports

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

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 vector as a platform for gene therapy delivery

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

Liver Expression of a MiniATP7B Gene Results in Long‐Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice

(2019) Oihana Murillo et al.   HEPATOLOGY

Onasemnogene Abeparvovec: First Global Approval

(2019) Sheridan M. Hoy   DRUGS

Gene therapy for glycogen storage diseases

(2019) Priya S Kishnani et al.   HUMAN MOLECULAR GENETICS

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

(2019) Albert M. Maguire et al.   OPHTHALMOLOGY

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

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

High‐throughput compound screen reveals mTOR inhibitors as potential therapeutics to reduce (auto)antibody production by human plasma cells

(2019) Paul Tuijnenburg et al.   EUROPEAN JOURNAL OF 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

Optimized Adeno-Associated Virus-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques

(2018) Jenny Greig 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

B Cell–Intrinsic mTORC1 Promotes Germinal Center–Defining Transcription Factor Gene Expression, Somatic Hypermutation, and Memory B Cell Generation in Humoral Immunity

(2018) Ariel L. Raybuck et al.   JOURNAL OF IMMUNOLOGY

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

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

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

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

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

(2018) Amine Meliani et al.   Nature Communications

Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone

(2017) Megan L. Cramer et al.   HUMAN GENE THERAPY

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice

(2017) Jenny A. Greig et al.   HUMAN GENE THERAPY

Germinal Center Selection and Affinity Maturation Require Dynamic Regulation of mTORC1 Kinase

(2017) Jonatan Ersching et al.   IMMUNITY

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

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

(2017) Marc Tardieu et al.   LANCET NEUROLOGY

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

Combination therapy for inhibitor reversal in haemophilia A using monoclonal anti-CD20 and rapamycin

(2017) Alexandra Sherman et al.   THROMBOSIS AND HAEMOSTASIS

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

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

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

AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees

(2016) Roberto Calcedo et al.   Human Gene Therapy Clinical Development

Controlling Toxicity of Peptide-Drug Conjugates by Different Chemical Linker Structures

(2015) David Böhme et al.   ChemMedChem

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

(2015) Manuela Corti et al.   Human Gene Therapy Clinical Development

Transient Low-Dose Methotrexate Generates B Regulatory Cells That Mediate Antigen-Specific Tolerance to Alglucosidase Alfa

(2014) Marguerite S. Joly et al.   JOURNAL OF IMMUNOLOGY

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

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

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

(2013) J. McIntosh et al.   BLOOD

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

(2013) Barbara K. Smith et al.   HUMAN GENE THERAPY

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

B Cell-Specific Deficiencies in mTOR Limit Humoral Immune Responses

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

Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates

(2013) Louis G. Chicoine et al.   MOLECULAR THERAPY

Absolute Determination of Single-Stranded and Self-Complementary Adeno-Associated Viral Vector Genome Titers by Droplet Digital PCR

(2013) Martin Lock et al.   Human Gene Therapy Methods

The Plasma Cell Signature in Autoimmune Disease

(2013) Katie Streicher et al.   Arthritis & Rheumatology

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

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

(2012) Yoav H. Messinger et al.   GENETICS IN MEDICINE

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

(2012) Seppo Ylä-Herttuala   MOLECULAR THERAPY

Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses

(2012) Agnieszka Ciesielska 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

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

(2011) Marianna Hösel et al.   HEPATOLOGY

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

Quantitative proteomic analysis of cyclosporine-induced toxicity in a human kidney cell line and comparison with tacrolimus

(2011) F. Lamoureux et al.   Journal of Proteomics

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

Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B

(2011) Sushrusha Nayak et al.   Frontiers in Microbiology

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

(2010) A. Joseph et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

(2010) Martin Lock et al.   HUMAN 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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

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

(2009) Greg M. Delgoffe et al.   IMMUNITY

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

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

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

Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells

(2009) S. NAYAK et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

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

(2009) Lili Wang et al.   MOLECULAR THERAPY

Elimination of Antibodies to Recombinant Enzyme in Pompe's Disease

(2009) Nancy J. Mendelsohn et al.   NEW ENGLAND JOURNAL OF MEDICINE