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Nature Medicine, ISSN 1078-8956, 02/2010, Volume 16, Issue 2, pp. 198 - 204
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2016, Volume 138, Issue 1, pp. 219 - 228.e9
Background Defects in phagocytic nicotinamide adenine dinucleotide phosphate oxidase 2 (NOX2) function cause chronic granulomatous disease (CGD), a primary... 
Allergy and Immunology | dysfunctional hematopoiesis | anakinra | Chronic granulomatous disease | cell cycle | hematopoietic stem cell | competitive repopulation assay | gene therapy | engraftment defect | IL-1β | hyperinflammation | NADPH OXIDASE | CGD | ACTIVATION | STERILE INFLAMMATION | IL-1 | IL-1 beta | NLRP3 INFLAMMASOME | IMMUNOLOGY | ALLERGY | INFLAMMATORY MANIFESTATIONS | IN-VIVO | GENE-THERAPY | MICE | Cell Count | Humans | Granulomatous Disease, Chronic - metabolism | Child, Preschool | Case-Control Studies | Young Adult | Inflammation Mediators - metabolism | Adult | Cell Differentiation | Colony-Forming Units Assay | Child | Disease Models, Animal | Hematopoietic Stem Cells - drug effects | Cytokines - metabolism | Signal Transduction | Hematopoietic Stem Cell Transplantation | Graft Survival | Immunophenotyping | Mice, Transgenic | Hematopoietic Stem Cells - metabolism | Granulomatous Disease, Chronic - etiology | Phenotype | Animals | Models, Biological | Hematopoietic Stem Cells - cytology | Adolescent | Biomarkers | Mice | Cytokines - pharmacology | Oxidases | Phosphates | Niacinamide | Purines | Stem cell research | Care and treatment | Immunodeficiency | Hematopoietic stem cells | Analysis | Competition | Cytokines | Granulocytes | Rodents | Stem cells | Inflammation | Laboratory animals | Experiments | Chemokines
Journal Article
Molecular Therapy, ISSN 1525-0016, 01/2015, Volume 23, Issue 1, pp. 63 - 70
Journal Article
Journal Article
Blood, ISSN 0006-4971, 03/2018, Volume 131, Issue 9, pp. 1022 - 1031
Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment,... 
CLINICAL-TRIAL | EFFICACY | REFERENCE-STANDARD MATERIAL | SAFETY | LIVER | PADUA | HEMATOLOGY | TYPE-2 | Thrombosis and Hemostasis
Journal Article
Blood, ISSN 0006-4971, 04/2003, Volume 101, Issue 8, pp. 3164 - 3173
The receptor tyrosine kinase Flt3 is expressed and functionally important in early myeloid progenitor cells and in the majority of acute myeloid leukeimia... 
BINDING-PROTEIN-ALPHA | INTERNAL TANDEM DUPLICATION | C/EBP-ALPHA | CONSTITUTIVE ACTIVATION | RISK GROUP | 32D CELLS | C-MYC | GRANULOCYTIC DIFFERENTIATION | CELL-LINES | HEMATOLOGY | SIGNAL TRANSDUCER | Oligonucleotide Array Sequence Analysis | Humans | Leukemia, Myeloid - genetics | Protein-Serine-Threonine Kinases | Receptor Protein-Tyrosine Kinases - physiology | Recombinant Fusion Proteins - physiology | fms-Like Tyrosine Kinase 3 | Gene Expression Profiling | Tandem Repeat Sequences | Proto-Oncogene Proteins - biosynthesis | Interleukin-3 - pharmacology | Leukemia, Myeloid - pathology | CCAAT-Enhancer-Binding Protein-alpha - biosynthesis | Transfection | Trans-Activators - genetics | Transcription, Genetic | Trans-Activators - biosynthesis | Neoplasm Proteins - genetics | Cell Line - metabolism | Acute Disease | Neoplasm Proteins - biosynthesis | Gene Expression Regulation | Genes, Synthetic | Proto-Oncogene Proteins - genetics | Hematopoietic Stem Cells - metabolism | Gene Expression Regulation, Leukemic | Reverse Transcriptase Polymerase Chain Reaction | Protein Isoforms - physiology | Animals | Receptor Protein-Tyrosine Kinases - genetics | Proto-Oncogene Proteins - physiology | CCAAT-Enhancer-Binding Protein-alpha - genetics | Myeloid Cells - metabolism | Recombinant Fusion Proteins - genetics | Computer Systems | Mice | Cluster Analysis
Journal Article
Journal Article
Blood, ISSN 0006-4971, 07/2005, Volume 106, Issue 1, pp. 265 - 273
Journal Article