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Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2015, Volume 136, Issue 3, pp. 692 - 702.e2
Background Wiskott-Aldrich syndrome (WAS) is a severe X-linked immunodeficiency characterized by microthrombocytopenia, eczema, recurrent infections, and... 
Allergy and Immunology | Wiskott-Aldrich syndrome | primary immunodeficiency | B cell | gene therapy | lentiviral vector | RHEUMATOID-ARTHRITIS | SYSTEMIC-LUPUS-ERYTHEMATOSUS | COMPLEMENT | ADHERENCE TEST | ACTIN POLYMERIZATION | SYNDROME PROTEIN | IMMUNOLOGY | TRANSPLANTATION | PRIMARY IMMUNODEFICIENCY DISEASES | ALLERGY | ACTIVATING FACTOR | LYMPHOCYTES | Wiskott-Aldrich Syndrome Protein - genetics | Bone Marrow - immunology | Humans | Recombinant Fusion Proteins - therapeutic use | Child, Preschool | Infant | Male | Transplantation, Autologous | Gene Expression Profiling | Autoantibodies - biosynthesis | Wiskott-Aldrich Syndrome - genetics | Wiskott-Aldrich Syndrome - therapy | Hematopoietic Stem Cells - immunology | Bone Marrow - metabolism | B-Lymphocyte Subsets - immunology | Lentivirus - genetics | Wiskott-Aldrich Syndrome - pathology | Child | Immunoglobulins - biosynthesis | Gene Expression | Wiskott-Aldrich Syndrome - immunology | Transduction, Genetic | B-Lymphocyte Subsets - pathology | Hematopoietic Stem Cell Transplantation | Immunophenotyping | Hematopoietic Stem Cells - metabolism | B-Cell Activating Factor - metabolism | B-Lymphocyte Subsets - metabolism | Wiskott-Aldrich Syndrome Protein - immunology | B-Cell Activating Factor - genetics | Bone Marrow - pathology | Transplantation Conditioning | Genetic Vectors | Genetic Therapy - methods | Autoimmunity | Care and treatment | Genetic vectors | Genes | Immunodeficiency | Genetic research | Lymphomas | Transplantation | Gene therapy | Health aspects | Hematopoietic stem cells | Bone marrow | Immunoglobulins | Patients | Immune Deficiencies, Infection, and Systemic Immune Disorders | WASp, Wiskott-Aldrich syndrome protein | WAS, Wiskott-Aldrich syndrome | GT, Gene therapy | PB, Peripheral blood | IVIg, Intravenous immunoglobulin | VCN, Vector copy number | HSC, Hematopoietic stem cell | HD, Healthy donor | BAFF, B cell–activating factor | BM, Bone marrow | SDF-1α, Stromal cell–derived factor 1α
Journal Article
The Journal of Allergy and Clinical Immunology, ISSN 0091-6749, 09/2019, Volume 144, Issue 3, pp. 825 - 838
Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe... 
Wiskott-Aldrich syndrome | X-linked thrombocytopenia | gene therapy | platelets | CLEARANCE | CD40 LIGAND | CELL TRANSPLANTATION | SYNDROME PROTEIN | NEUTROPHILS | SPLENECTOMY | IMMUNOLOGY | ACTIVATED PLATELETS | ALLERGY | MUTATIONS | EXPRESSION | Genetic research | Care and treatment | Genetic aspects | Gene therapy | Analysis | Flow cytometry | Pediatrics | Transplants & implants | Medical services | Clinical trials | Agglomeration | Coexistence | Bleeding | Defects | Proteins | Genotype & phenotype | Signal transduction | Protein composition | Restoration | Blood platelets | Ultrastructure | Thrombocytopenia | Phenotypes | Immune reconstitution | Electron microscopy | Patients | Microscopy | Protein expression | Software | Mutation | Platelets | FERMT3, Fermitin family homolog 3 | TEM, Transmission electron microscopy | WAS, Wiskott-Aldrich syndrome | HMGB1, High-mobility group box 1 | δ-g, Electron-dense granule | GPX1, Glutathione peroxidase 1 | FU, Follow-up | sCD62P, Soluble P-selectin | sCD40L, Soluble CD40 ligand | BAFF, B cell–activating factor | CD62P, P-selectin | HSCT, Hematopoietic stem cell transplantation | WASp, Wiskott-Aldrich syndrome protein | MFI, Mean fluorescence intensity | LV, Lentivirus | CT, Closure time | GT, Gene therapy | OCS, Open canalicular system | PRP, Platelet-rich plasma | vWF, von Willebrand factor | STAT3, Signal transducer and activator of transcription 3 | HD, Healthy donor | ADP, Adenosine diphosphate | ROS, Reactive oxygen species | XLT, X-linked thrombocytopenia
Journal Article
Human Vaccines & Immunotherapeutics, ISSN 2164-5515, 02/2015, Volume 11, Issue 2, pp. 450 - 457
Journal Article
Autophagy, ISSN 1554-8627, 12/2014, Volume 10, Issue 12, pp. 2109 - 2121
Journal Article
Autoimmunity, ISSN 0891-6934, 9/2004, Volume 37, Issue 6-7, pp. 495 - 501
Journal Article
Nature Medicine, ISSN 1078-8956, 2014, Volume 20, Issue 8, pp. 936 - 941
Journal Article
Nature Medicine, ISSN 1078-8956, 11/2012, Volume 18, Issue 11, pp. 1682 - 1687
Journal Article
PLoS Pathogens, ISSN 1553-7366, 12/2011, Volume 7, Issue 12, p. e1002425
Myeloid blood cells are largely resistant to infection with human immunodeficiency virus type 1 (HIV-1). Recently, it was reported that Vpx from HIV-2/SIVsm... 
DOMAIN | HUMAN DENDRITIC CELLS | INNATE IMMUNE-RESPONSE | MONOCYTES | MICROBIOLOGY | RESTRICTION | SAMHD1 | REPLICATION | VIROLOGY | IMMUNODEFICIENCY-VIRUS TYPE-1 | EXPRESSION | PARASITOLOGY | VPX | Immunoprecipitation | Humans | SAM Domain and HD Domain-Containing Protein 1 | Immunoblotting | Mutation, Missense | Lipopolysaccharide Receptors - metabolism | Tandem Mass Spectrometry | Flow Cytometry | Transfection | Nervous System Malformations - metabolism | Autoimmune Diseases of the Nervous System - virology | Autoimmune Diseases of the Nervous System - genetics | Autoimmune Diseases of the Nervous System - metabolism | Nervous System Malformations - genetics | HIV-1 - metabolism | Genetic Predisposition to Disease - genetics | Enzyme-Linked Immunosorbent Assay | Cell Separation | HIV Infections - genetics | Myeloid Cells - virology | Monomeric GTP-Binding Proteins - genetics | Reverse Transcriptase Polymerase Chain Reaction | Monomeric GTP-Binding Proteins - deficiency | Nervous System Malformations - virology | Microscopy, Confocal | Myeloid Cells - metabolism | Prevention | Aicardi syndrome | Disease susceptibility | Genetic aspects | Research | T cells | Health aspects | HIV infection | Risk factors | Proteins | Peptides | Microscopy | Genes | Interferon | Mutation | Autoimmune diseases | Mass spectrometry | Patients | Viral infections
Journal Article