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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
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2008, Volume 122, Issue 6, pp. 1105 - 1112.e1
Background Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is an autoimmune genetic disorder caused by mutation of the forkhead... 
Allergy and Immunology | regulatory T cells | transcription factor | Immune dysregulation | autoimmunity | immunosuppressive treatment | polyendocrinopathy | enteropathy | X-linked syndrome | FOXP3 | bone marrow transplantation | Forkhead Transcription Factors - immunology | Polyendocrinopathies, Autoimmune - diagnosis | Humans | Polyendocrinopathies, Autoimmune - therapy | Male | Intestinal Diseases - immunology | DNA Mutational Analysis - methods | Mutation - immunology | Genetic Diseases, X-Linked - genetics | Polyendocrinopathies, Autoimmune - genetics | Immunologic Deficiency Syndromes - diagnosis | Immunologic Deficiency Syndromes - immunology | Immunologic Deficiency Syndromes - therapy | Gene Expression Regulation - genetics | Gene Expression Regulation - immunology | Intestinal Diseases - genetics | Genotype | Protein Structure, Tertiary - genetics | Forkhead Transcription Factors - genetics | Genetic Diseases, X-Linked - diagnosis | Syndrome | Polyendocrinopathies, Autoimmune - immunology | Intestinal Diseases - therapy | Genetic Diseases, X-Linked - immunology | Intestinal Diseases - diagnosis | Phenotype | Immunologic Deficiency Syndromes - genetics | Genetic Diseases, X-Linked - therapy | Genetic disorders | Stem cells | Bone marrow | Cystic fibrosis | Transplantation | Diabetes | Gene therapy | Antitank weapons | Proteins | Genotype & phenotype | Transplants & implants | Disease | Genes | Protein expression | Mutation | Patients | Age | Deoxyribonucleic acid--DNA
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2011, Volume 127, Issue 6, pp. 1368 - 1375.e8
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2014, Volume 133, Issue 3, pp. 799 - 806.e10
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2010, Volume 125, Issue 3, pp. 727 - 736
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2011, Volume 127, Issue 6, pp. 1376 - 1384.e5
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2010, Volume 126, Issue 6, pp. 1242 - 1251
Background Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) is a primary immunodeficiency with autoimmunity caused by mutations in... 
Allergy and Immunology | IPEX | autoimmunity | T regulatory cells | FOXP3 | tolerance | IL-17 | ACTIVATION | ROR-GAMMA-T | TRANSCRIPTION | IMMUNOLOGY | SCURFIN | GROWTH-FACTOR-BETA | TARGET GENES | ALLERGY | FOXP3 GENE | MUTATIONS | EXPRESSION | Forkhead Transcription Factors - immunology | T-Lymphocyte Subsets - immunology | Immunologic Deficiency Syndromes - pathology | T-Lymphocytes, Regulatory - metabolism | Cell Proliferation | Humans | Cell Transdifferentiation - genetics | T-Lymphocytes, Regulatory - pathology | T-Lymphocytes, Regulatory - immunology | Young Adult | Point Mutation - genetics | Forkhead Transcription Factors - metabolism | Mutant Proteins - immunology | Immunologic Deficiency Syndromes - immunology | Child | Cytokines - genetics | Polyendocrinopathies, Autoimmune | Cytokines - metabolism | Transgenes - genetics | Cells, Cultured | Mutant Proteins - genetics | Mutant Proteins - metabolism | Forkhead Transcription Factors - genetics | Immunosuppression | T-Lymphocyte Subsets - pathology | T-Lymphocyte Subsets - metabolism | Immunologic Deficiency Syndromes - physiopathology | Adolescent | Immunologic Deficiency Syndromes - genetics | CD4 Antigens - biosynthesis | Autoimmunity | Immunodeficiency | Stem cells | Genetic research | Fluorescence | Nerve growth factor | Children | T cells | Health aspects | Transcription factors | Disease | Lymphocytes | Rodents | Eczema | Diarrhea | Software | Mutation | Experiments
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2010, Volume 125, Issue 2, pp. 439 - 448.e8
Journal Article
Lancet, The, ISSN 0140-6736, 2016, Volume 388, Issue 10043, pp. 476 - 487
Journal Article