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1. Full Text Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome
by Hacein-Bey Abina, Salima and Gaspar, H. Bobby and Blondeau, Johanna and Caccavelli, Laure and Charrier, Sabine and Buckland, Karen and Picard, Capucine and Six, Emmanuelle and Himoudi, Nourredine and Gilmour, Kimberly and McNicol, Anne-Marie and Hara, Havinder and Xu-Bayford, Jinhua and Rivat, Christine and Touzot, Fabien and Mavilio, Fulvio and Lim, Annick and Treluyer, Jean-Marc and Héritier, Sébastien and Lefrère, Francois and Magalon, Jeremy and Pengue-Koyi, Isabelle and Honnet, Géraldine and Blanche, Stéphane and Sherman, Eric A and Male, Frances and Berry, Charles and Malani, Nirav and Bushman, Frederic D and Fischer, Alain and Thrasher, Adrian J and Galy, Anne and Cavazzana, Marina
JAMA : the journal of the American Medical Association, ISSN 0098-7484, 04/2015, Volume 313, Issue 15, pp. 1550 - 1563
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Severity of Illness Index | Gene Expression | Wiskott-Aldrich Syndrome - immunology | Humans | Child, Preschool | Infant | Male | Lentivirus | Wiskott-Aldrich Syndrome - genetics | Feasibility Studies | Wiskott-Aldrich Syndrome - therapy | Adolescent | Hematopoietic Stem Cell Transplantation - adverse effects | Genetic Vectors | Wiskott-Aldrich Syndrome Protein Family - genetics | Child | Infant, Newborn | Genetic Therapy - adverse effects | Wiskott-Aldrich syndrome | Treatment outcome | Usage | Care and treatment | Analysis | Gene therapy | Risk factors | Patient safety | Autoimmune diseases | Clinical outcomes | Index Medicus | Abridged Index Medicus | Genetic Therapy | Hematopoietic Stem Cell Transplantation | Life Sciences | Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome Protein Family
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2. Full Text B-cell reconstitution after lentiviral vector–mediated gene therapy in patients with Wiskott-Aldrich syndrome
by Castiello, Maria Carmina, PhD and Scaramuzza, Samantha, PhD and Pala, Francesca, MS and Ferrua, Francesca, MD and Uva, Paolo, PhD and Brigida, Immacolata, PhD and Sereni, Lucia, MS and van der Burg, Mirjam, PhD and Ottaviano, Giorgio, MD and Albert, Michael H., MD and Grazia Roncarolo, Maria, MD and Naldini, Luigi, MD, PhD and Aiuti, Alessandro, MD, PhD and Villa, Anna, MD and Bosticardo, Marita, PhD
Journal of allergy and clinical immunology, ISSN 0091-6749, 2015, Volume 136, Issue 3, pp. 692 - 702.e2
Allergy and Immunology | Wiskott-Aldrich syndrome | primary immunodeficiency | B cell | gene therapy | lentiviral vector | Allergy | Immunology | Life Sciences & Biomedicine | Science & Technology | 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 | Index Medicus | Abridged Index Medicus | 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
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3. Full Text IL-2 induces a WAVE2-dependent pathway for actin reorganization that enables WASp-independent human NK cell function
by Orange, Jordan S and Roy-Ghanta, Sumita and Mace, Emily M and Maru, Saumya and Rak, Gregory D and Sanborn, Keri B and Fasth, Anders and Saltzman, Rushani and Paisley, Allison and Monaco-Shawver, Linda and Banerjee, Pinaki P and Pandey, Rahul and Sahlgrenska akademin and Institute of Clinical Sciences, Department of Pediatrics and Göteborgs universitet and Gothenburg University and Institutionen för kliniska vetenskaper, Avdelningen för pediatrik and Sahlgrenska Academy
The Journal of clinical investigation, ISSN 0021-9738, 04/2011, Volume 121, Issue 4, pp. 1535 - 1548
Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Cell Line | Wiskott-Aldrich Syndrome - immunology | Wiskott-Aldrich Syndrome Protein Family - metabolism | Humans | Actins - metabolism | Wiskott-Aldrich Syndrome Protein - antagonists & inhibitors | Wiskott-Aldrich Syndrome Protein Family - immunology | Signal Transduction - immunology | Interleukin-2 - immunology | Wiskott-Aldrich Syndrome Protein - immunology | Interleukin-2 - pharmacology | Killer Cells, Natural - immunology | Wiskott-Aldrich Syndrome Protein - metabolism | Killer Cells, Natural - drug effects | Killer Cells, Natural - metabolism | In Vitro Techniques | Cytotoxicity, Immunologic | Wiskott-Aldrich Syndrome - metabolism | Wiskott-Aldrich syndrome | Actin | Disease susceptibility | Genetic aspects | Research | Properties | Health aspects | Index Medicus | Abridged Index Medicus | primary immunodeficiency | Pediatrics | Wiskott Aldrich syndrome | Pediatrik
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4. Full Text Mycolactone activation of Wiskott-Aldrich syndrome proteins underpins Buruli ulcer formation
by Guenin-Macé, Laure and Veyron-Churlet, Romain and Thoulouze, Maria-Isabel and Romet-Lemonne, Guillaume and Hong, Hui and Leadlay, Peter F and Danckaert, Anne and Ruf, Marie-Thérèse and Mostowy, Serge and Zurzolo, Chiara and Bousso, Philippe and Chrétien, Fabrice and Carlier, Marie-France and Demangel, Caroline
The Journal of clinical investigation, ISSN 0021-9738, 04/2013, Volume 123, Issue 4, pp. 1501 - 1512
Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Humans | Protein Multimerization | Actins - metabolism | Molecular Sequence Data | Actin-Related Protein 2-3 Complex - metabolism | Mycobacterium ulcerans | Cell Nucleus - metabolism | Buruli Ulcer - microbiology | Wiskott-Aldrich Syndrome Protein, Neuronal - antagonists & inhibitors | Actins - chemistry | Macrolides - pharmacology | Buruli Ulcer - pathology | Wiskott-Aldrich Syndrome Protein, Neuronal - metabolism | Amino Acid Sequence | Epidermis - pathology | Actin Cytoskeleton - metabolism | Wiskott-Aldrich Syndrome Protein Family - metabolism | Epidermis - drug effects | Mice, Inbred C57BL | Wiskott-Aldrich Syndrome Protein Family - antagonists & inhibitors | Cell Adhesion | Protein Transport | Animals | Keratinocytes - drug effects | Bacterial Toxins - pharmacology | Keratinocytes - metabolism | Mice | HeLa Cells | Carbazoles - pharmacology | Propanolamines - pharmacology | Buruli Ulcer - metabolism | Cell Movement | Wiskott-Aldrich syndrome | Physiological aspects | Development and progression | Buruli ulcer | Health aspects | Mycotoxins | Index Medicus | Abridged Index Medicus | Cellular Biology | Life Sciences | Bacterial Toxins | Wiskott-Aldrich Syndrome Protein Family | Actin Cytoskeleton | Wiskott-Aldrich Syndrome Protein, Neuronal | Macrolides | Keratinocytes | Actins | Epidermis | Actin-Related Protein 2-3 Complex | Carbazoles | Buruli Ulcer | Cell Nucleus | Propanolamines
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5. Full Text Wiskott-Aldrich syndrome protein-mediated actin dynamics control type-I interferon production in plasmacytoid dendritic cells
by Prete, Francesca and Catucci, Marco and Labrada, Mayrel and Gobessi, Stefania and Castiello, Maria Carmina and Bonomi, Elisa and Aiuti, Alessano and Vermi, William and Cancrini, Caterina and Metin, Ayse and Hambleton, Sophie and Bredius, Robbert and Notarangelo, Lucia and Burg, Mirjam and Kalinke, Ulrich and Villa, A.M and Benvenuti, Federica
The Journal of experimental medicine, ISSN 0022-1007, 02/2013, Volume 210, Issue 2, pp. 355 - 374
cytokine production | signal transduction | gene silencing | plasmacytoid dendritic cell | disease predisposition | protein function | autoimmune disease | animal experiment | article | actin polymerization | recurrent infection | mouse | Wiskott Aldrich syndrome | autoimmunity | controlled study | hematopoietic cell | innate immunity | nonhuman | priority journal | Immunology | Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Autoimmunity | Wiskott-Aldrich Syndrome Protein - genetics | Wiskott-Aldrich Syndrome Protein - deficiency | Dendritic Cells - immunology | Humans | Actins - metabolism | Dendritic Cells - pathology | Male | RNA, Messenger - metabolism | Wiskott-Aldrich Syndrome - genetics | Interferon Type I - biosynthesis | Endocytosis | Interferon-alpha - genetics | Base Sequence | Wiskott-Aldrich Syndrome Protein - metabolism | Female | Wiskott-Aldrich Syndrome - pathology | Receptor, Interferon alpha-beta - genetics | Dendritic Cells - metabolism | Disease Models, Animal | Wiskott-Aldrich Syndrome - metabolism | Wiskott-Aldrich Syndrome - immunology | Signal Transduction | Interferon-alpha - biosynthesis | Receptor, Interferon alpha-beta - deficiency | Mice, Inbred C57BL | RNA, Messenger - genetics | Immunity, Innate | Mice, Knockout | Actins - antagonists & inhibitors | Animals | Interferon Type I - genetics | Mice | Toll-Like Receptor 9 - metabolism | Index Medicus
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6. Full Text Hierarchical Regulation of WASP/WAVE Proteins
by Padrick, Shae B and Cheng, Hui-Chun and Ismail, Ayman M and Panchal, Sanjay C and Doolittle, Lynda K and Kim, Soyeon and Skehan, Brian M and Umetani, Junko and Brautigam, Chad A and Leong, John M and Rosen, Michael K
Molecular cell, ISSN 1097-2765, 11/2008, Volume 32, Issue 3, pp. 426 - 438
CELLBIO | PROTEINS | Biochemistry & Molecular Biology | Life Sciences & Biomedicine | Science & Technology | Cell Biology | Wiskott-Aldrich Syndrome Protein - genetics | Wiskott-Aldrich Syndrome Protein Family - metabolism | Allosteric Regulation | Humans | Actins - metabolism | Homeostasis | Wiskott-Aldrich Syndrome Protein Family - chemistry | Wiskott-Aldrich Syndrome - genetics | Protein Folding | Image Processing, Computer-Assisted | Wiskott-Aldrich Syndrome Protein - metabolism | Protein Conformation | Cell Membrane - metabolism | Kinetics | Wiskott-Aldrich Syndrome Protein Family - genetics | Binding Sites | Dimerization | Wiskott-Aldrich Syndrome - metabolism | Muscle proteins | Actin | Index Medicus
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7. Full Text Wiskott–Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans
by Castiello, Maria Carmina and Bosticardo, Marita and Pala, Francesca and Catucci, Marco and Chamberlain, Nicolas and van Zelm, Menno C and Driessen, Gertjan J and Pac, Malgorzata and Bernatowska, Ewa and Scaramuzza, Samantha and Aiuti, Alessandro and Sauer, Aisha V and Traggiai, Elisabetta and Meffre, Eric and Villa, Anna and van der Burg, Mirjam
Journal of autoimmunity, ISSN 0896-8411, 2013, Volume 50, Issue 100, pp. 42 - 50
Allergy and Immunology | Autoimmunity | B-cell activating factor (BAFF) | Immunoglobulin repertoire | B lymphocyte | Wiskott–Aldrich Syndrome | Primary immunodeficiency | Wiskott-Aldrich Syndrome | Wiskott-aldrich syndrome | Life Sciences & Biomedicine | Immunology | Science & Technology | Fundamental and applied biological sciences. Psychology | Hematologic and hematopoietic diseases | Fundamental immunology | Immunopathology | Biological and medical sciences | Medical sciences | Immunodeficiencies. Immunoglobulinopathies | Immunodeficiencies | Platelet diseases and coagulopathies | Wiskott-Aldrich Syndrome Protein - genetics | Wiskott-Aldrich Syndrome Protein - deficiency | Bone Marrow - immunology | Humans | Receptors, Complement 3d - immunology | Wiskott-Aldrich Syndrome - genetics | Chemokine CXCL12 - genetics | Disease Susceptibility - immunology | Receptors, Complement 3d - genetics | B-Cell Activating Factor - immunology | Wiskott-Aldrich Syndrome - pathology | Cell Differentiation | B-Lymphocytes - pathology | Immunoglobulin Heavy Chains - immunology | Chemokine CXCL12 - immunology | Gene Expression | Wiskott-Aldrich Syndrome - immunology | Homeostasis - immunology | B-Lymphocytes - immunology | Wiskott-Aldrich Syndrome Protein - immunology | B-Cell Activating Factor - genetics | Bone Marrow - pathology | Immunologic Memory | B-Cell Activating Factor - blood | Immunoglobulin Heavy Chains - genetics | Cell Movement | Wiskott-Aldrich syndrome | Immunoglobulins | Analysis | Mortality | Immunodeficiency | Homeostasis | Cells | Index Medicus
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8. Full Text A novel primary human immunodeficiency due to deficiency in the WASP-interacting protein WIP
by Lanzi, Gaetana and Moratto, Daniele and Vairo, Donatella and Masneri, Stefania and Delmonte, Ottavia and Paganini, Tiziana and Parolini, Silvia and Tabellini, Giovanna and Mazza, Cinzia and Savoldi, Gianfranco and Montin, Davide and Martino, Silvana and