Related references
Note: Only part of the references are listed.BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression
Barbara A. Konkle et al.
BLOOD (2021)
Journey to the Center of the Cell: Tracing the Path of AAV Transduction
Bijay P. Dhungel et al.
TRENDS IN MOLECULAR MEDICINE (2021)
Overcoming innate immune barriers that impede AAV gene therapy vectors
Manish Muhuri et al.
JOURNAL OF CLINICAL INVESTIGATION (2021)
Proteasome Inhibitor Drugs
Lloyd D. Fricker
ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY, VOL 60 (2020)
Rabbit ATG/ATLG in preventing graft-versus-host disease after allogeneic stem cell transplantation: consensus-based recommendations by an international expert panel
Francesca Bonifazi et al.
BONE MARROW TRANSPLANTATION (2020)
Mechanisms of action of hydroxychloroquine and chloroquine: implications for rheumatology
Eva Schrezenmeier et al.
NATURE REVIEWS RHEUMATOLOGY (2020)
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Giuseppe Ronzitti et al.
FRONTIERS IN IMMUNOLOGY (2020)
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
Ang Li et al.
MOLECULAR THERAPY (2020)
rAAV8 and rAAV9-Mediated Long-Term Muscle Transduction with Tacrolimus (FK506) in Non-Human Primates
Akiko Ishii et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Christian Leborgne et al.
NATURE MEDICINE (2020)
Re: Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy by Wilson and Flotte
Perry B. Shieh et al.
HUMAN GENE THERAPY (2020)
B Cell Depletion Eliminates FVIII Memory B Cells and Enhances AAV8-coF8 Immune Tolerance Induction When Combined With Rapamycin
Moanaro Biswas et al.
FRONTIERS IN IMMUNOLOGY (2020)
Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates
Benjamin J. Samelson-Jones et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Immune Response Mechanisms against AAV Vectors in Animal Models
Ashley T. Martino et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Lindsey A. George et al.
MOLECULAR THERAPY (2020)
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
Christian Mueller et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
Juliette Hordeaux et al.
SCIENCE TRANSLATIONAL MEDICINE (2020)
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
K. John Pasi et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Identification of preexisting adaptive immunity to Cas9 proteins in humans
Carsten T. Charlesworth et al.
NATURE MEDICINE (2019)
Adeno-associated virus vector as a platform for gene therapy delivery
Dan Wang et al.
NATURE REVIEWS DRUG DISCOVERY (2019)
Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the ND4 Gene A Secondary Analysis of a Phase 1/2 Clinical Trial
Celine Bouquet et al.
JAMA OPHTHALMOLOGY (2019)
Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer
Sylvie Da Rocha et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy
Barry J. Byrne et al.
ANNALS OF TRANSLATIONAL MEDICINE (2019)
Liver induced transgene tolerance with AAV vectors
Geoffrey D. Keeler et al.
CELLULAR IMMUNOLOGY (2019)
Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues
Laurel C. Chandler et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design
Pierce J. Ogden et al.
SCIENCE (2019)
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
Morgan L. Maeder et al.
NATURE MEDICINE (2019)
Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle
Jerome Poupiot et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Safety, immunogenicity, pharmacokinetics, and efficacy of degradation of anti-HLA antibodies by IdeS (imlifidase) in chronic kidney disease patients
Tomas Lorant et al.
AMERICAN JOURNAL OF TRANSPLANTATION (2018)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Christian Hinderer et al.
HUMAN GENE THERAPY (2018)
Gene Therapy with Regulatory T Cells: A Beneficial Alliance
Moanaro Biswas et al.
FRONTIERS IN IMMUNOLOGY (2018)
Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction
Zachary Fitzpatrick et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques
Juliette Hordeaux et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Glucocorticoid-Induced Osteoporosis
Lenore Buckley et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
Amine Meliani et al.
NATURE COMMUNICATIONS (2018)
Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction
Wenwei Shao et al.
JCI INSIGHT (2018)
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B
Wolfgang Miesbach et al.
BLOOD (2018)
Safety of proteasome inhibitors for treatment of multiple myeloma
Danielle Schlafer et al.
EXPERT OPINION ON DRUG SAFETY (2017)
Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
Celine Vandamme et al.
HUMAN GENE THERAPY (2017)
Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone
Megan L. Cramer et al.
HUMAN GENE THERAPY (2017)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al.
LANCET (2017)
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency
Christian Mueller et al.
MOLECULAR THERAPY (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
L. A. George et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency
Roberto Calcedo et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)
Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion
Longping Victor Tse et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
Manuela Corti et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2017)
Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting
Victoria M. Velazquez et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Michael F. Naso et al.
BIODRUGS (2017)
Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis
Daniel Gaudet et al.
HUMAN GENE THERAPY (2016)
Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning
Manuela Corti et al.
HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2015)
AAV capsid CD8+T-cell epitopes are highly conserved across AAV serotypes
Daniel J. Hui et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2015)
Safety and Effectiveness of Mycophenolate in Systemic Sclerosis. A Systematic Review
Mohammed A. Omair et al.
PLOS ONE (2015)
Clinical Use of Proteasome Inhibitors in the Treatment of Multiple Myeloma
Noah M. Merin et al.
PHARMACEUTICALS (2015)
The incidence, management, and evolution of rapamycin-related side effects in kidney transplant recipients
Jacobien Verhave et al.
CLINICAL TRANSPLANTATION (2014)
Effect of bortezomib on the efficacy of AAV9.SERCA2a treatment to preserve cardiac function in a rat pressure-overload model of heart failure
A. H. Chaanine et al.
GENE THERAPY (2014)
mTOR kinase inhibitors promote antibody class switching via mTORC2 inhibition
Jose J. Limon et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)
The efficacy and safety of daclizumab and its potential role in the treatment of multiple sclerosis
Ron Milo
THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS (2014)
Immune responses to AAV-vectors, the Glybera example from bencn to bedside
Valerie Ferreira et al.
FRONTIERS IN IMMUNOLOGY (2014)
Development of gene transfer for induction of antigen-specific tolerance
Brandon K. Sack et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2014)
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
Federico Mingozzi et al.
BLOOD (2013)
Chemotherapy induced DNA damage response Convergence of drugs and pathways
Derek Woods et al.
CANCER BIOLOGY & THERAPY (2013)
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
F. Mingozzi et al.
GENE THERAPY (2013)
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
D. Gaudet et al.
GENE THERAPY (2013)
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression
Christian Mueller et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
Calcineurin Inhibitors: 40 Years Later, Can't Live Without
Jamil R. Azzi et al.
JOURNAL OF IMMUNOLOGY (2013)
PharmGKB summary: cyclosporine and tacrolimus pathways
Julia M. Barbarino et al.
PHARMACOGENETICS AND GENOMICS (2013)
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Federico Mingozzi et al.
SCIENCE TRANSLATIONAL MEDICINE (2013)
A practical guide to the monitoring and management of the complications of systemic corticosteroid therapy
Dora Liu et al.
ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY (2013)
Differing Effects of Rapamycin or Calcineurin Inhibitor on T-Regulatory Cells in Pediatric Liver and Kidney Transplant Recipients
T. Akimova et al.
AMERICAN JOURNAL OF TRANSPLANTATION (2012)
Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine
J. H. McIntosh et al.
GENE THERAPY (2012)
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
C. Li et al.
GENE THERAPY (2012)
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates
Carmen Unzu et al.
JOURNAL OF TRANSLATIONAL MEDICINE (2012)
Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B
Federico Mingozzi et al.
MOLECULAR THERAPY (2012)
In Vitro Effects of Cyclosporine A and Tacrolimus on Regulatory T-Cell Proliferation and Function
Celine Miroux et al.
TRANSPLANTATION (2012)
Clinical review: Serious adverse events associated with the use of rituximab - a critical care perspective
Pashtoon M. Kasi et al.
CRITICAL CARE (2012)
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
Ashley T. Martino et al.
BLOOD (2011)
Mycophenolate Mofetil Impairs Transduction of Single-Stranded Adeno-Associated Viral Vectors
Paula S. Montenegro-Miranda et al.
HUMAN GENE THERAPY (2011)
The anti-inflammatory and immunosuppressive effects of glucocorticoids, recent developments and mechanistic insights
Agnes E. Coutinho et al.
MOLECULAR AND CELLULAR ENDOCRINOLOGY (2011)
A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8
Virginie Monteilhet et al.
MOLECULAR THERAPY (2011)
Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B
Amit C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2011)
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
Sylvie Boutin et al.
HUMAN GENE THERAPY (2010)
Sirolimus for calcineurin inhibitors in organ transplantation: contra
Paolo Cravedi et al.
KIDNEY INTERNATIONAL (2010)
Proteasome Inhibitors Decrease AAV2 Capsid-derived Peptide Epitope Presentation on MHC Class I Following Transduction
Jonathan D. Finn et al.
MOLECULAR THERAPY (2010)
Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application
Paul E. Monahan et al.
MOLECULAR THERAPY (2010)
Calcineurin Inhibitor Nephrotoxicity
Maarten Naesens et al.
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY (2009)
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
Jiangao Zhu et al.
JOURNAL OF CLINICAL INVESTIGATION (2009)
Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses
Roberto Calcedo et al.
JOURNAL OF INFECTIOUS DISEASES (2009)
Processing of recombinant AAV genomes occurs in specific nuclear structures that overlap with foci of DNA-damage-response proteins
Tiziana Cervelli et al.
JOURNAL OF CELL SCIENCE (2008)
Differential impact of mammalian target of rapamycin inhibition on CD4(+)CD25(+)Foxp3(+) regulatory T cells compared with conventional CD4(+) T cells
Robert Zeiser et al.
BLOOD (2008)
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
Federico Mingozzi et al.
BLOOD (2007)
Mechanisms of action of antithymocyte globulin: T-cell depletion and beyond
M. Mohty
LEUKEMIA (2007)
CD8+ T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi et al.
NATURE MEDICINE (2007)
Proteasomal chymotrypsin-like peptidase activity is required for essential functions of human monocyte-derived dendritic cells
Cord Naujokat et al.
IMMUNOLOGY (2007)
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
Haiyan Jiang et al.
BLOOD (2006)
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
CS Manno et al.
NATURE MEDICINE (2006)
Rituximab (monoclonal anti-CD20 antibody): mechanisms of action and resistance
MR Smith
ONCOGENE (2003)
Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9
PA Fields et al.
MOLECULAR THERAPY (2001)
Adverse gastrointestinal effects of mycophenolate mofetil - Aetiology, incidence and management
M Behrend
DRUG SAFETY (2001)