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1. Full Text Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
by Gaspar, H Bobby and Parsley, Kathryn L and Howe, Steven and King, Doug and Gilmour, Kimberly C and Sinclair, Joanna and Brouns, Gaby and Schmidt, Manfred and Von Kalle, Christof and Barington, Torben and Jakobsen, Marianne A and Christensen, Hans O and Al Ghonaium, Abdulaziz and White, Harry N and Smith, John L and Levinsky, Roland J and Ali, Robin R and Kinnon, Christine and Thrasher, Adrian J
The Lancet (British edition), ISSN 0140-6736, 12/2004, Volume 364, Issue 9452, pp. 2181 - 2187
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Biological and medical sciences | General aspects | Medical sciences | Antigens, CD34 - analysis | Humans | Child, Preschool | Infant | Immunity | Lymphocyte Culture Test, Mixed | Receptors, Interleukin-7 - genetics | Bone Marrow Cells - immunology | Bone Marrow Transplantation | Immunoglobulins - blood | Genetic Diseases, X-Linked - genetics | Gene Transfer Techniques | Transduction, Genetic | Lymphocyte Activation | Severe Combined Immunodeficiency - therapy | Severe Combined Immunodeficiency - immunology | Severe Combined Immunodeficiency - genetics | Gammaretrovirus | T-Lymphocytes - immunology | Interleukin Receptor Common gamma Subunit | Mutation | Genetic Vectors | Genetic Diseases, X-Linked - therapy | Genetic Therapy - adverse effects | Genetic Therapy - methods | Gene therapy | Research | Bone marrow | Clinical trials | Transplants & implants | Autoimmune diseases | Severe combined immunodeficiency | Leukemia | Bone marrow transplantation | Recovery of function | Viruses | Transplantation | Lymphocytes T | T-cell receptor | Killer cells | Immunology | Restoration | Lymphocytes | Immunity (humoral) | Children | CD34 antigen | Antigens | Autografts | Immunoglobulins | Cytokines | Patients | Morbidity | Chemotherapy | Lymphocytes B | Stem cells | Histocompatibility antigen HLA | Index Medicus | Abridged Index Medicus
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2. Full Text Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
by Howe, Steven J and Mansour, Marc R and Schwarzwaelder, Kerstin and Bartholomae, Cynthia and Hubank, Michael and Kempski, Helena and Brugman, Martijn H and Pike-Overzet, Karin and Chatters, Stephen J and De Ridder, Dick and Gilmour, Kimberly C and Adams, Stuart and Thornhill, Susannah I and Parsley, Kathryn L and Staal, Frank J. T and Gale, Rosemary E and Linch, David C and Bayford, Jinhua and Brown, Lucie and Quaye, Michelle and Kinnon, Christine and Ancliff, Philip and Webb, David K and Schmidt, Manfred and Von Kalle, Christof and Gaspar, H. Bobby and Thrasher, Adrian J
The Journal of clinical investigation, ISSN 0021-9738, 09/2008, Volume 118, Issue 9, pp. 3143 - 3150
Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Follow-Up Studies | LIM Domain Proteins | Humans | Cyclin-Dependent Kinase Inhibitor p16 - genetics | Severe Combined Immunodeficiency - therapy | Infant | Male | DNA-Binding Proteins - genetics | Chromosomes, Human, X | Receptors, Interleukin-2 - genetics | Severe Combined Immunodeficiency - complications | Adaptor Proteins, Signal Transducing | Mutagenesis | Models, Biological | Metalloproteins - genetics | Precursor T-Cell Lymphoblastic Leukemia-Lymphoma - etiology | Antineoplastic Agents - pharmacology | Proto-Oncogene Proteins | Mutation | Receptor, Notch1 - genetics | Precursor T-Cell Lymphoblastic Leukemia-Lymphoma - therapy | Genetic Therapy - adverse effects | Genetic Therapy - methods | Precursor T-Cell Lymphoblastic Leukemia-Lymphoma - complications | Complications and side effects | Care and treatment | Severe combined immunodeficiency | Genetic aspects | Research | Gene therapy | Methods | Risk factors | Index Medicus | Abridged Index Medicus
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3. Full Text Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells
by Santilli, Giorgia and Almarza, Elena and Brendel, Christian and Choi, Uimook and Beilin, Chiara and Blundell, Michael P and Haria, Sneha and Parsley, Kathryn L and Kinnon, Christine and Malech, Harry L and Bueren, Juan A and Grez, Manuel and Thrasher, Adrian J
Molecular therapy, ISSN 1525-0016, 01/2011, Volume 19, Issue 1, pp. 122 - 132
Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Genetic Vectors - administration & dosage | Humans | Myeloid Cells - physiology | Granulomatous Disease, Chronic - genetics | Granulomatous Disease, Chronic - metabolism | NADPH Oxidases - metabolism | Molecular Sequence Data | Stem Cells - metabolism | Recombinant Fusion Proteins - metabolism | DNA Copy Number Variations | Lentivirus - metabolism | Cell Differentiation - genetics | Genes, X-Linked | Retroviridae - metabolism | Retroviridae - genetics | Base Sequence | NADPH Oxidases - genetics | Lentivirus - genetics | Granulocytes - metabolism | Terminal Repeat Sequences | Transgenes | Binding Sites | CCAAT-Enhancer-Binding Proteins - metabolism | Proto-Oncogene Proteins - metabolism | Recombinant Fusion Proteins - biosynthesis | Promoter Regions, Genetic | Cathepsin G - genetics | Proto-Oncogene Proteins c-fes - genetics | Gene Expression Regulation | Mutagenesis - genetics | Genetic Vectors - metabolism | Spleen Focus-Forming Viruses - metabolism | Granulomatous Disease, Chronic - therapy | Hematopoietic Stem Cells - metabolism | Spleen Focus-Forming Viruses - genetics | Genetic Vectors - genetics | Animals | Enhancer Elements, Genetic | Cell Line, Tumor | Myeloid Cells - metabolism | Recombinant Fusion Proteins - genetics | Trans-Activators - metabolism | Granulomatous Disease, Chronic - enzymology | Mice | Receptors, Immunologic - genetics | Receptors, Immunologic - metabolism | Genetic Therapy - methods | Index Medicus | Original
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4. Full Text Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency
by Thornhill, Susannah I and Schambach, Axel and Howe, Steven J and Ulaganathan, Meera and Grassman, Elke and Williams, David and Schiedlmeier, Bernhard and Sebire, Neil J and Gaspar, H Bobby and Kinnon, Christine and Baum, Christopher and Thrasher, Adrian J
Molecular therapy, ISSN 1525-0016, 03/2008, Volume 16, Issue 3, pp. 590 - 598
Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Humans | X-Linked Combined Immunodeficiency Diseases - genetics | X-Linked Combined Immunodeficiency Diseases - immunology | T-Lymphocytes - metabolism | Polymerase Chain Reaction | Killer Cells, Natural - immunology | Cell Differentiation | B-Lymphocytes - metabolism | Disease Models, Animal | Cell Line | B-Lymphocytes - cytology | Enzyme-Linked Immunosorbent Assay | Mice, Inbred C57BL | Cells, Cultured | X-Linked Combined Immunodeficiency Diseases - therapy | Genetic Vectors - genetics | Killer Cells, Natural - cytology | Blotting, Northern | Animals | B-Lymphocytes - immunology | Gammaretrovirus - genetics | T-Lymphocytes - cytology | T-Lymphocytes - immunology | Mice | Models, Genetic | Killer Cells, Natural - metabolism | Genetic Therapy - methods | Index Medicus
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5. Full Text Immunodeficiency: Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction
by Gaspar, H. Bobby and Cooray, Samantha and Gilmour, Kimberly C and Parsley, Kathryn L and Zhang, Fang and Adams, Stuart and Bjorkegren, Emma and Bayford, Jinhua and Brown, Lucinda and Davies, E. Graham and Veys, Paul and Fairbanks, Lynette and Bordon, Victoria and Petropolou, Theoni and Kinnon, Christine and Thrasher, Adrian J
Science translational medicine, ISSN 1946-6234, 08/2011, Volume 3, Issue 97
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6. Full Text Immunodeficiency: Long-term persistence of a polyclonal t cell repertoire after gene therapy for X-linked severe combined immunodeficiency
by Gaspar, H. Bobby and Cooray, Samantha and Gilmour, Kimberly C and Parsley, Kathryn L and Adams, Stuart and Howe, Steven J and Al Ghonaium, Abdulaziz and Bayford, Jinhua and Brown, Lucinda and Davies, E. Graham and Kinnon, Christine and Thrasher, Adrian J
Science translational medicine, ISSN 1946-6234, 08/2011, Volume 3, Issue 97
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7. Full Text Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning
by Gaspar, H. Bobby and Bjorkegren, Emma and Parsley, Kate and Gilmour, Kimberly C and King, Doug and Sinclair, Joanna and Zhang, Fang and Giannakopoulos, Aris and Adams, Stuart and Fairbanks, Lynette D and Gaspar, Jane and Henderson, Lesley and Xu-Bayford, Jin Hua and Davies, E. Graham and Veys, Paul A and Kinnon, Christine and Thrasher, Adrian J
Molecular therapy, ISSN 1525-0016, 10/2006, Volume 14, Issue 4, pp. 505 - 513
gene therapy | retroviral vector | SCID | adenosine deaminase | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Genetic Therapy | Severe Combined Immunodeficiency - enzymology | Follow-Up Studies | Polyethylene Glycols | Adenosine Deaminase - genetics | Adenosine Deaminase - metabolism | Humans | Cells, Cultured | Child, Preschool | Hematopoietic Stem Cell Transplantation | Severe Combined Immunodeficiency - therapy | Infant | Male | Gene Dosage | Severe Combined Immunodeficiency - immunology | Genetic Vectors - genetics | Cell Lineage | Severe Combined Immunodeficiency - genetics | T-Lymphocytes - cytology | T-Lymphocytes - immunology | Transplantation Conditioning | Hematopoietic Stem Cells - enzymology | Adenosine Deaminase - immunology | Retrotransposons | Enzymes | Chemotherapy | Polyethylene | Metabolites | Polyethylene glycol | Bone marrow | Transplantation | Gene therapy | Hematopoietic stem cells | Cancer | Index Medicus
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8. Full Text In-home assessment of either topical fluralaner or topical selamectin for flea control in naturally infested cats in West Central Florida, USA
by Dryden, Michael W and Canfield, Michael S and Bocon, Cara and Phan, Letitia and Niedfeldt, Emily and Kinnon, Amanda and Warcholek, Stanislaw A and Smith, Vicki and Bress, Todd S and Smith, Nicole and Heaney, Kathleen and Royal, Christine and Normile, Dorothy and Armstrong, Robert and Sun, Fangshi
Parasites & vectors, ISSN 1756-3305, 07/2018, Volume 11, Issue 1, pp. 422 - 422
Fluralaner | Cats | Ctenocephalides felis felis | Dogs | Sarolaner | Pruritus | Flea control | Selamectin | Flea allergy dermatitis | Parasitology | Life Sciences & Biomedicine | Science & Technology | Tropical Medicine | Insecticides | Control | Comparative analysis | Fleas | Veterinarians | Populations | Households | Group homes | Topical application | Lesions | Dermatitis | Index Medicus
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9. Full Text Bone morphogenetic protein-4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA
: Correspondence
by Abeyewickreme, Anil and Thrasher, Adrian J and Kinnon, Christine
British journal of haematology, ISSN 0007-1048, 12/2011, Volume 155, Issue 5, pp. 638 - 641
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10. Full Text Bone morphogenetic protein‐4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA
by Abeyewickreme, Anil and Thrasher, Adrian J and Kinnon, Christine
British journal of haematology, ISSN 0007-1048, 12/2011, Volume 155, Issue 5, pp. 638 - 641
bone morphogenetic protein | embryonic stem cells | haematopoiesis | gene expression | short hairpin RNA | Bone morphogenetic protein | Embryonic stem cells | Gene expression | Short hairpin RNA | Haematopoiesis | Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Cell Differentiation - genetics | RNA, Small Interfering - genetics | Up-Regulation | Animals | Embryonic Stem Cells - cytology | Embryonic Stem Cells - drug effects | Cell Differentiation - drug effects | Bone Morphogenetic Protein 4 - genetics | Humans | Mice | RNA, Small Interfering - administration & dosage | Bone Morphogenetic Protein 4 - pharmacology | Bone morphogenetic proteins | RNA | Genes | Index Medicus
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11. Full Text Codon optimization of human factor VIII cDNAs leads to high-level expression
by Ward, Natalie J and Buckley, Suzanne M. K and Waddington, Simon N and VandenDriessche, Thierry and Chuah, Marinee K. L and Nathwani, Amit C and McIntosh, Jenny and Tuddenham, Edward G. D and Kinnon, Christine and Thrasher, Adrian J and McVey, John H
Blood, ISSN 0006-4971, 01/2011, Volume 117, Issue 3, pp. 798 - 807
Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Biological and medical sciences | Medical sciences | Injections, Intravenous | Genetic Vectors - administration & dosage | Humans | Molecular Sequence Data | Male | Mice, 129 Strain | Promoter Regions, Genetic - genetics | HEK293 Cells | Female | Lentivirus - genetics | Codon - genetics | Animals, Newborn | Amino Acid Sequence | Gene Expression | Enzyme-Linked Immunosorbent Assay | Factor VIII - genetics | Spleen Focus-Forming Viruses - genetics | Hemophilia A - therapy | Reverse Transcriptase Polymerase Chain Reaction | Hemophilia A - blood | Genetic Vectors - genetics | Mice, Knockout | Sequence Homology, Amino Acid | Animals | Hemophilia A - genetics | Mice | Factor VIII - metabolism | Mutation | Genetic Therapy - methods | Amino acids | Index Medicus | Abridged Index Medicus
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12. Full Text Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia
by Ancliff, Phil J and Blundell, Michael P and Cory, Giles O and Calle, Yolanda and Worth, Austen and Kempski, Helena and Burns, Siobhan and Jones, Gareth E and Sinclair, Jo and Kinnon, Christine and Hann, Ian M and Gale, Rosemary E and Linch, David C and Thrasher, Adrian J
Blood, ISSN 0006-4971, 10/2006, Volume 108, Issue 7, pp. 2182 - 2189
Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Other diseases. Hematologic involvement in other diseases | Immunopathology | Biological and medical sciences | Medical sciences | Immunodeficiencies. Immunoglobulinopathies | Immunodeficiencies | Leukocyte Elastase - metabolism | Wiskott-Aldrich Syndrome Protein - genetics | Cell Proliferation | Chlorocebus aethiops | Bone Marrow Cells - cytology | Neutropenia - metabolism | Humans | Child, Preschool | Male | Lymphocytes - cytology | Neutropenia - congenital | Animals | U937 Cells | Wiskott-Aldrich Syndrome Protein - metabolism | Mutation | Neutropenia - genetics | COS Cells | Child | Bone Marrow Cells - metabolism | Index Medicus | Abridged Index Medicus
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