相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression
Barbara A. Konkle et al.
BLOOD (2021)
Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses
Ying Kai Chan et al.
SCIENCE TRANSLATIONAL MEDICINE (2021)
Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles
Petr O. Ilyinskii et al.
SCIENCE ADVANCES (2021)
Sirolimus Pharmacokinetics Variability Points to the Relevance of Therapeutic Drug Monitoring in Pediatric Oncology
Amelia-Naomi Sabo et al.
PHARMACEUTICS (2021)
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Helena Costa Verdera et al.
MOLECULAR THERAPY (2020)
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?
J. Fraser Wright
MOLECULAR THERAPY (2020)
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
Berangere Bertin et al.
SCIENTIFIC REPORTS (2020)
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Christian Leborgne et al.
NATURE MEDICINE (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)
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)
Quantification of CpG Motifs in rAAVGenomes: Avoiding the Toll
J. Fraser Wright
MOLECULAR THERAPY (2020)
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme
Zachary C. Elmore et al.
JCI INSIGHT (2020)
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
K. John Pasi et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Activation of NF-κB in B cell receptor signaling through Bruton's tyrosine kinase-dependent phosphorylation of IκB-α
Marilena Pontoriero et al.
JOURNAL OF MOLECULAR MEDICINE-JMM (2019)
Complexity of immune responses to AAV transgene products - Example of factor IX
Roland W. Herzog
CELLULAR IMMUNOLOGY (2019)
Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis
Geoffrey D. Keeler et al.
MOLECULAR THERAPY (2018)
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
Amine Meliani et al.
NATURE COMMUNICATIONS (2018)
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B
Wolfgang Miesbach et al.
BLOOD (2018)
Ibrutinib may impair serological responses to influenza vaccination
Abby P. Douglas et al.
HAEMATOLOGICA (2017)
Autophagy Determines Efficiency of Liver-Directed Gene Therapy With Adeno-Associated Viral Vectors
Marianna Hoesel et al.
HEPATOLOGY (2017)
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
L. A. George et al.
NEW ENGLAND JOURNAL OF MEDICINE (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)
Ibrutinib suppresses alloantibody responses in a mouse model of allosensitization
Irene Kim et al.
TRANSPLANT IMMUNOLOGY (2017)
Ibrutinib may impair serological responses to influenza vaccination
Abby P. Douglas et al.
HAEMATOLOGICA (2017)
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
Amine Meliani et al.
BLOOD ADVANCES (2017)
Lymphangioleiomyomatosis
Simon R. Johnson et al.
CLINICS IN CHEST MEDICINE (2016)
Everolimus and sirolimus in transplantation-related but different
Jost Klawitter et al.
EXPERT OPINION ON DRUG SAFETY (2015)
Clinically-relevant cyclosporin and rapamycin concentrations enhance regulatory T cell function to a similar extent but with different mechanisms: An in-vitro study in healthy humans
Daniela Fanigliulo et al.
INTERNATIONAL IMMUNOPHARMACOLOGY (2015)
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
Jerry R. Mendell et al.
MOLECULAR THERAPY (2015)
High-throughput combinatorial screening identifies drugs that cooperate with ibrutinib to kill activated B-cell-like diffuse large B-cell lymphoma cells
Lesley A. Mathews Griner et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)
B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study
M. Corti et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2014)
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
Jenny McIntosh et al.
BLOOD (2013)
B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease
Melissa E. Elder et al.
JOURNAL OF PEDIATRICS (2013)
Mapping the Structural Determinants Responsible for Enhanced T Cell Activation to the Immunogenic Adeno-Associated Virus Capsid from Isolate Rhesus 32.33
Lauren E. Mays et al.
JOURNAL OF VIROLOGY (2013)
Inhibitors of BTK and ITK: State of the New Drugs for Cancer, Autoimmunity and Inflammatory Diseases
L. Vargas et al.
SCANDINAVIAN JOURNAL OF IMMUNOLOGY (2013)
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Federico Mingozzi et al.
SCIENCE TRANSLATIONAL MEDICINE (2013)
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
C. Li et al.
GENE THERAPY (2012)
Self-complementary AAVs Induce More Potent Transgene Product-specific Immune Responses Compared to a Single-stranded Genome
TeLang Wu et al.
MOLECULAR THERAPY (2012)
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
Dawn E. Bowles et al.
MOLECULAR THERAPY (2012)
Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results
Terence R. Flotte et al.
HUMAN GENE THERAPY (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)
Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates
Hua Li 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)
Prevention and reversal of antibody responses against factor IX in gene therapy for hemophilia B
Sushrusha Nayak et al.
FRONTIERS IN MICROBIOLOGY (2011)
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
Glenn P. Niemeyer et al.
BLOOD (2009)
Cytotoxic-T-Lymphocyte-Mediated Elimination of Target Cells Transduced with Engineered Adeno-Associated Virus Type 2 Vector In Vivo
Chengwen Li et al.
JOURNAL OF VIROLOGY (2009)
A Preclinical Animal Model to Assess the Effect of Pre-existing Immunity on AAV-mediated Gene Transfer
Hua Li et al.
MOLECULAR THERAPY (2009)
mTOR regulates memory CD8 T-cell differentiation
Koichi Araki et al.
NATURE (2009)
Tolerance Induction to Cytoplasmic β-Galactosidase by Hepatic AAV Gene Transfer - Implications for Antigen Presentation and Immunotoxicity
Ashley T. Martino et al.
PLOS ONE (2009)
Safety and efficacy of gene transfer for Leber's congenital amaurosis
Albert M. Maguire et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Delays in maturation among adolescents with hemophilia and a history of inhibitors
Sharyne M. Donfield et al.
BLOOD (2007)
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)
CD8+ T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi et al.
NATURE MEDICINE (2007)
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)
Regulation of B-cell fate by antigen-receptor signals
H Niiro et al.
NATURE REVIEWS IMMUNOLOGY (2002)
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)
分享论文
