Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study

[1]  D. Richman,et al.  Precision targeting of autoantigen-specific B cells in muscle-specific tyrosine kinase myasthenia gravis with chimeric autoantibody receptor T cells , 2023, Nature Biotechnology.

[2]  W. Wang,et al.  Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results , 2023, Journal of the Neurological Sciences.

[3]  D. Stroncek,et al.  Genome-wide profiling of retroviral DNA integration and its effect on clinical pre-infusion CAR T-cell products , 2022, Journal of Translational Medicine.

[4]  E. Manasanch,et al.  The role of soluble B cell maturation antigen as a biomarker in multiple myeloma , 2022, Leukemia & lymphoma.

[5]  A. Ekici,et al.  Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus , 2022, Nature Medicine.

[6]  M. V. D. van den Brink,et al.  CAR-T cell therapy-related cytokine release syndrome and therapeutic response is modulated by the gut microbiome in hematologic malignancies , 2022, Nature Communications.

[7]  C. Ramos,et al.  Preparing for CAR T cell therapy: patient selection, bridging therapies and lymphodepletion , 2022, Nature Reviews Clinical Oncology.

[8]  Mark M. Davis,et al.  KIR+CD8+ T cells suppress pathogenic T cells and are active in autoimmune diseases and COVID-19 , 2022, Science.

[9]  C. Meisel,et al.  Daratumumab for treatment‐refractory antibody‐mediated diseases in neurology , 2022, European journal of neurology.

[10]  N. Silvestri,et al.  Utilization of MG‐ADL in myasthenia gravis clinical research and care , 2022, Muscle & nerve.

[11]  R. Conwit,et al.  Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study. , 2021, Neurology.

[12]  B. Xiao,et al.  Single-cell RNA-Seq reveals transcriptional heterogeneity and immune subtypes associated with disease activity in human myasthenia gravis , 2021, Cell discovery.

[13]  J. Statland,et al.  Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial , 2021, The Lancet Neurology.

[14]  C. Zografou,et al.  Short- and Long-Lived Autoantibody-Secreting Cells in Autoimmune Neurological Disorders , 2021, Frontiers in Immunology.

[15]  M. Maus,et al.  Recent advances and discoveries in the mechanisms and functions of CAR T cells , 2021, Nature Reviews Cancer.

[16]  Shigeaki Suzuki,et al.  Roles of cytokines and T cells in the pathogenesis of myasthenia gravis , 2020, Clinical and experimental immunology.

[17]  S. Gambhir,et al.  Molecular Imaging of Chimeric Antigen Receptor T Cells by ICOS-ImmunoPET , 2020, Clinical Cancer Research.

[18]  K. Anderson,et al.  Preclinical evaluation of CD8+ anti-BCMA mRNA CAR T-cells for treatment of multiple myeloma , 2020, Leukemia.

[19]  P. van Damme,et al.  ‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab , 2020, Journal of Neurology.

[20]  Anurag K Singh,et al.  CAR T cells: continuation in a revolution of immunotherapy. , 2020, The Lancet. Oncology.

[21]  M. Benatar,et al.  Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis , 2020, JAMA neurology.

[22]  J. Kochenderfer,et al.  Recent advances in CAR T-cell toxicity: Mechanisms, manifestations and management. , 2019, Blood reviews.

[23]  Z. Siddiqi,et al.  Therapies Directed Against B-Cells and Downstream Effectors in Generalized Autoimmune Myasthenia Gravis: Current Status , 2019, Drugs.

[24]  Michel Sadelain,et al.  Chimeric Antigen Receptor Therapy. , 2018, The New England journal of medicine.

[25]  D. Weissman,et al.  mRNA vaccines — a new era in vaccinology , 2018, Nature Reviews Drug Discovery.

[26]  K. O’Connor,et al.  Mechanisms underlying B cell immune dysregulation and autoantibody production in MuSK myasthenia gravis , 2018, Annals of the New York Academy of Sciences.

[27]  G. L. Masson,et al.  Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. , 2017, The Lancet. Neurology.

[28]  Daniel Li,et al.  Kinetics and biomarkers of severe cytokine release syndrome after CD19 chimeric antigen receptor-modified T-cell therapy. , 2017, Blood.

[29]  K. O’Connor,et al.  Autoantibody-producing plasmablasts after B cell depletion identified in muscle-specific kinase myasthenia gravis. , 2017, JCI insight.

[30]  R. Hohlfeld,et al.  Human Plasmacytoid Dendritic Cells Display and Shed B Cell Maturation Antigen upon TLR Engagement , 2017, The Journal of Immunology.

[31]  M. Conaway,et al.  International clinimetric evaluation of the MG‐QOL15, resulting in slight revision and subsequent validation of the MG‐QOL15r , 2016, Muscle & nerve.

[32]  A. Vincent,et al.  Use of cell-based assays in myasthenia gravis and other antibody-mediated diseases , 2015, Experimental Neurology.

[33]  B. Levine,et al.  T Cells Expressing Chimeric Antigen Receptors Can Cause Anaphylaxis in Humans , 2013, Cancer Immunology Research.

[34]  M. Raffeld,et al.  B-cell Maturation Antigen Is a Promising Target for Adoptive T-cell Therapy of Multiple Myeloma , 2013, Clinical Cancer Research.

[35]  V. Bril,et al.  The Quantitative Myasthenia Gravis Score: Comparison With Clinical, Electrophysiological, and Laboratory Markers , 2012, Journal of clinical neuromuscular disease.

[36]  Tomoki Ito,et al.  Neutrophils Activate Plasmacytoid Dendritic Cells by Releasing Self-DNA–Peptide Complexes in Systemic Lupus Erythematosus , 2011, Science Translational Medicine.

[37]  M. Conaway,et al.  The MG Composite , 2010, Neurology.

[38]  Y. Monden,et al.  Acetylcholine receptor antibody production by bone marrow cells in a patient with myasthenia gravis , 1985, Neurology.

[39]  S. Shapiro,et al.  An Analysis of Variance Test for Normality (Complete Samples) , 1965 .

[40]  D. Darling,et al.  A Test of Goodness of Fit , 1954 .

[41]  Nils E Gilhus.  Myasthenia Gravis. , 2016, The New England journal of medicine.