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    Wyświetlanie 1-16 z 16
    Tytuł:
    Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials.
    Autorzy:
    Segura EER; Molecular Biology Interdepartmental Doctoral Program, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.
    Ayoub PG; Department of Molecular & Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.
    Hart KL; Department of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.
    Kohn DB; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.; Department of Pediatrics (Hematology/Oncology), David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.; Eli and Edythe Broad Center for Stem Cell Research and Regenerative Medicine, University of California, Los Angeles, CA 90095, USA.
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    Źródło:
    Viruses [Viruses] 2023 Mar 09; Vol. 15 (3). Date of Electronic Publication: 2023 Mar 09.
    Typ publikacji:
    Journal Article; Review; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    beta-Thalassemia*/genetics
    beta-Thalassemia*/therapy
    Hematopoietic Stem Cell Transplantation*/methods
    Hemoglobinopathies*/genetics
    Hemoglobinopathies*/therapy
    Anemia, Sickle Cell*/genetics
    Anemia, Sickle Cell*/therapy
    Humans ; Genetic Vectors/genetics ; Genetic Therapy/methods ; beta-Globins/genetics
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknie Pełny tekstSzczegóły
    Tytuł:
    Correction to: Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization.
    Autorzy:
    Hazim RA; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Karumbayaram S; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Jiang M; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Dimashkie A; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.
    Lopes VS; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Li D; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.; Department of Molecular Cell and Developmental Biology, Los Angeles, CA, USA.
    Burgess BL; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Vijayaraj P; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.; Department of Pediatrics, David Geffen School of Medicine, Los Angeles, CA, USA.
    Alva-Ornelas JA; Department of Population Sciences, City of Hope National Medical Center, Duarte, CA, USA.
    Zack JA; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.; Department of Medicine, David Geffen School of Medicine, Los Angeles, CA, USA.
    Kohn DB; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.; Department of Pediatrics, David Geffen School of Medicine, Los Angeles, CA, USA.
    Gomperts BN; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.; Department of Pediatrics, David Geffen School of Medicine, Los Angeles, CA, USA.
    Pyle AD; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Lowry WE; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.; Department of Molecular Cell and Developmental Biology, Los Angeles, CA, USA.
    Williams DS; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA. .; Department of Neurobiology, David Geffen School of Medicine, Los Angeles, CA, USA. .; Molecular Biology Institute, Los Angeles, CA, USA. .; Brain Research Institute, University of California, Los Angeles, CA, USA. .
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    Źródło:
    Stem cell research & therapy [Stem Cell Res Ther] 2019 Feb 12; Vol. 10 (1), pp. 52. Date of Electronic Publication: 2019 Feb 12.
    Typ publikacji:
    Published Erratum
    Pełny tekst  Link otwiera się w nowym oknie Pełny tekstSzczegóły
    Tytuł:
    T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.
    Autorzy:
    Clarke EL; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Connell AJ; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Six E; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, France.; Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France.
    Kadry NA; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Abbas AA; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Hwang Y; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Everett JK; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Hofstaedter CE; Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
    Marsh R; Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, 45229-3039, USA.
    Armant M; Boston Children's Hospital, Karp 08125.3, 300 Longwood Avenue, Boston, MA, 02115, USA.
    Kelsen J; Division of Gastroenterology, Hepatology, and Nutrition, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
    Notarangelo LD; Laboratory of Host Defenses, Laboratory of Clinical Infectious Diseases, Immune Deficiency Genetics Section, NIAID, NIH, Bethesda, MD, USA.
    Collman RG; Department of Medicine, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA.
    Hacein-Bey-Abina S; Clinical Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, 78, r. du Général-Leclerc, 94270, Le-Kremlin-Bicêtre, France.; UTCBS CNRS UMR 8258, INSERM U1022, Faculté de Pharmacie de Paris, Université Paris Descartes, Sorbonne Paris Cité, Chimie Paris-Tech, 4 av. de l'observatoire, 75006, Paris, France.
    Kohn DB; Departments of Microbiology, Immunology & Molecular Genetics; and Pediatrics, University of California, Los Angeles, USA.
    Cavazzana M; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, France.; Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France.; Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM, Paris, France.
    Fischer A; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, France.; Laboratory of Human Lymphohematopoiesis, INSERM UMR 1163, Paris, France.; Pediatric Hemato-Immunology Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.; Collège de France, Paris, France.
    Williams DA; Boston Children's Hospital, Karp 08125.3, 300 Longwood Avenue, Boston, MA, 02115, USA.; Havard Stem Cell Institute, Harvard Medical School, Boston, MA, 02115, USA.
    Pai SY; Boston Children's Hospital, Karp 08125.3, 300 Longwood Avenue, Boston, MA, 02115, USA.
    Bushman FD; Department of Microbiology, University of Pennsylvania School of Medicine, 3610 Hamilton Walk, Philadelphia, PA, 19104-6076, USA. .
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    Źródło:
    Genome medicine [Genome Med] 2018 Sep 28; Vol. 10 (1), pp. 70. Date of Electronic Publication: 2018 Sep 28.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Genetic Therapy*
    Microbiota*
    T-Lymphocytes/*immunology
    X-Linked Combined Immunodeficiency Diseases/*immunology
    X-Linked Combined Immunodeficiency Diseases/*therapy
    Cell Division ; Child, Preschool ; Complementarity Determining Regions/genetics ; Humans ; Receptors, Antigen, T-Cell, alpha-beta/metabolism ; X-Linked Combined Immunodeficiency Diseases/microbiology ; X-Linked Combined Immunodeficiency Diseases/virology
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknie Pełny tekstSzczegóły
    Tytuł:
    Gene therapy comes of age.
    Autorzy:
    Dunbar CE; Hematology Branch, National Heart, Lung and Blood Institute, Bethesda, MD, USA. .
    High KA; Spark Therapeutics, Philadelphia, PA, USA.
    Joung JK; Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.
    Kohn DB; David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
    Ozawa K; The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
    Sadelain M; Memorial Sloan Kettering Cancer Center, New York, NY, USA. .
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    Źródło:
    Science (New York, N.Y.) [Science] 2018 Jan 12; Vol. 359 (6372).
    Typ publikacji:
    Journal Article; Review
    MeSH Terms:
    Genetic Therapy*/adverse effects
    Animals ; Gene Editing ; Gene Transfer Techniques ; Genetic Diseases, Inborn/therapy ; Genetic Engineering ; Genetic Vectors ; Hematologic Diseases/therapy ; Humans ; Neoplasms/therapy ; Neuromuscular Diseases/therapy ; Translational Research, Biomedical
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknie Pełny tekstSzczegóły
    Tytuł:
    Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization.
    Autorzy:
    Hazim RA; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Karumbayaram S; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA. .; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA. .; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA. .
    Jiang M; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Dimashkie A; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.
    Lopes VS; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Li D; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.; Department of Molecular Cell and Developmental Biology, Los Angeles, CA, USA.
    Burgess BL; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA.
    Vijayaraj P; Department of Pediatrics, David Geffen School of Medicine, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Alva-Ornelas JA; Department of Population Sciences, City of Hope National Medical Center, Duarte, CA, USA.
    Zack JA; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.; Department of Medicine, David Geffen School of Medicine, Los Angeles, CA, USA.
    Kohn DB; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Department of Pediatrics, David Geffen School of Medicine, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Gomperts BN; Department of Pediatrics, David Geffen School of Medicine, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Pyle AD; Department of Microbiology Immunology and Molecular Genetics, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Lowry WE; Department of Molecular Cell and Developmental Biology, Los Angeles, CA, USA.; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, Los Angeles, CA, USA.; Jonsson Comprehensive Cancer Center, Los Angeles, CA, USA.
    Williams DS; Stein Eye Institute and Department of Ophthalmology, David Geffen School of Medicine at UCLA, 100 Stein Plaza, Los Angeles, CA, 90095, USA. .; Department of Neurobiology, David Geffen School of Medicine, Los Angeles, CA, USA. .; Molecular Biology Institute, Los Angeles, CA, USA. .; Brain Research Institute, University of California, Los Angeles, CA, USA. .
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    Źródło:
    Stem cell research & therapy [Stem Cell Res Ther] 2017 Oct 02; Vol. 8 (1), pp. 217. Date of Electronic Publication: 2017 Oct 02.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Cellular Reprogramming/*genetics
    Encephalitis Virus, Venezuelan Equine/*genetics
    Epithelial Cells/*drug effects
    Fibroblasts/*physiology
    Induced Pluripotent Stem Cells/*physiology
    Retinal Degeneration/*therapy
    Animals ; Cell Differentiation/drug effects ; Cell Polarity/drug effects ; Disease Models, Animal ; Encephalitis Virus, Venezuelan Equine/metabolism ; Epithelial Cells/cytology ; Epithelial Cells/physiology ; Epithelial Cells/transplantation ; Fibroblasts/cytology ; Genetic Vectors/chemistry ; Genetic Vectors/metabolism ; Humans ; Induced Pluripotent Stem Cells/cytology ; Induced Pluripotent Stem Cells/drug effects ; Injections, Intraocular ; Intercellular Signaling Peptides and Proteins/pharmacology ; Mice ; Mice, Inbred BALB C ; Mice, Knockout ; Primary Cell Culture ; Retinal Degeneration/pathology ; Retinal Degeneration/physiopathology ; Retinal Pigment Epithelium/cytology ; Retinal Pigment Epithelium/drug effects ; Retinal Pigment Epithelium/physiology ; Skin/cytology
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Impulse oscillometry identifies peripheral airway dysfunction in children with adenosine deaminase deficiency.
    Autorzy:
    Komarow HD; Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, NIH/NIAID/LAD/Bldg. 10, Room 1C129A1, 10 Center Drive, Bethesda, MD, 20892-1960, USA. .
    Sokolic R; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, 20892, USA. .
    Hershfield MS; Department of Biochemistry, Duke University School of Medicine, Durham, NC, USA. .
    Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, David Geffen School of Medicine, Los Angeles, CA, USA. .
    Young M; Clinical Research Directorate/Clinical Monitoring Research Program, Leidos Biomedical Research, Inc., Frederick National Laboratory for Clinical Research, Frederick, MD, 21702, USA. .
    Metcalfe DD; Laboratory of Allergic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, NIH/NIAID/LAD/Bldg. 10, Room 1C129A1, 10 Center Drive, Bethesda, MD, 20892-1960, USA. .
    Candotti F; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, 20892, USA. .; Division of Immunology and Allergy, University Hospital of Lausanne, CH-1101, Lausanne, Switzerland. .
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    Źródło:
    Orphanet journal of rare diseases [Orphanet J Rare Dis] 2015 Dec 18; Vol. 10, pp. 159. Date of Electronic Publication: 2015 Dec 18.
    Typ publikacji:
    Letter; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Adenosine Deaminase/*deficiency
    Agammaglobulinemia/*diagnosis
    Lung/*physiology
    Lung Diseases/*diagnosis
    Oscillometry/*methods
    Severe Combined Immunodeficiency/*diagnosis
    Adolescent ; Child ; Child, Preschool ; Female ; Humans ; Male ; Prospective Studies ; Spirometry
    SCR Disease Name:
    Severe combined immunodeficiency due to adenosine deaminase deficiency
    Raport
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    HIV eradication--from Berlin to Boston.
    Autorzy:
    Cannon PM; University of Southern California, Los Angeles, California, USA.
    Kohn DB; University of California Los Angeles, Los Angeles, California, USA.
    Kiem HP; Fred Hutchinson Cancer Research Center and University of Washington, Seattle, Washington, USA.
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    Źródło:
    Nature biotechnology [Nat Biotechnol] 2014 Apr; Vol. 32 (4), pp. 315-6.
    Typ publikacji:
    Letter; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    HIV Infections*/complications
    HIV Infections*/drug therapy
    HIV Infections*/virology
    HIV Seronegativity*
    Anti-Retroviral Agents/therapeutic use ; Berlin ; Boston ; HIV-1/drug effects ; Hematopoietic Stem Cell Transplantation ; Humans ; Leukemia/complications ; Leukemia/therapy ; Lymphoma/complications ; Lymphoma/therapy
    Raport
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Hematopoietic stem cells for cancer immunotherapy.
    Autorzy:
    Gschweng E; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, David Geffen School of Medicine, Los Angeles, CA, USA; Eli and Edythe Broad Center for Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, David Geffen School of Medicine, Los Angeles, CA, USA.
    De Oliveira S
    Kohn DB
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    Źródło:
    Immunological reviews [Immunol Rev] 2014 Jan; Vol. 257 (1), pp. 237-49.
    Typ publikacji:
    Journal Article; Review
    MeSH Terms:
    Immunotherapy*/methods
    Hematopoietic Stem Cells/*immunology
    Hematopoietic Stem Cells/*metabolism
    Neoplasms/*immunology
    Neoplasms/*therapy
    Animals ; Gene Transfer Techniques ; Genes, Transgenic, Suicide ; Genetic Engineering ; Hematopoietic Stem Cell Transplantation ; Humans ; Immunotherapy, Adoptive/methods ; Neoplasms/genetics ; Receptors, Antigen, T-Cell/genetics ; Receptors, Antigen, T-Cell/metabolism ; T-Lymphocytes/immunology ; T-Lymphocytes/metabolism
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status.
    Autorzy:
    Awe JP
    Lee PC
    Ramathal C
    Vega-Crespo A
    Durruthy-Durruthy J
    Cooper A
    Karumbayaram S
    Lowry WE
    Clark AT
    Zack JA
    Sebastiano V
    Kohn DB
    Pyle AD
    Martin MG
    Lipshutz GS
    Phelps PE
    Pera RA
    Byrne JA
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    Źródło:
    Stem cell research & therapy [Stem Cell Res Ther] 2013 Jul 26; Vol. 4 (4), pp. 87. Date of Electronic Publication: 2013 Jul 26.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Induced Pluripotent Stem Cells/*metabolism
    Animals ; Cell Differentiation ; Cellular Reprogramming ; Gene Expression ; Genomics ; Humans ; Mice ; Transgenes
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors.
    Autorzy:
    De Oliveira SN; Department of Pediatrics, University of California, Los Angeles (UCLA), 10833 Le Conte Avenue, A2-410 MDCC, MC 175217, Los Angeles, CA 90095-1752, USA. />Wang J
    Ryan C
    Morrison SL
    Kohn DB
    Hollis RP
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    Źródło:
    Journal of translational medicine [J Transl Med] 2013 Jan 29; Vol. 11, pp. 23. Date of Electronic Publication: 2013 Jan 29.
    Typ publikacji:
    Journal Article; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Antigens, CD19/*chemistry
    Immunoglobulin Fragments/*chemistry
    Receptors, Antigen/*chemistry
    Recombinant Fusion Proteins/*metabolism
    Animals ; Antibodies, Monoclonal/chemistry ; Cell Proliferation ; Cell Transplantation ; Dose-Response Relationship, Drug ; Enzyme-Linked Immunosorbent Assay ; Flow Cytometry ; HEK293 Cells ; Humans ; Immunoglobulin G/chemistry ; Leukocytes, Mononuclear/cytology ; Lymphocyte Activation ; Mice ; Mice, Inbred NOD ; Mice, SCID ; Protein Structure, Tertiary ; Recombinant Fusion Proteins/chemistry ; T-Lymphocytes/cytology
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo.
    Autorzy:
    Holt N; Keck School of Medicine of the University of Southern California, Los Angeles, California, USA.
    Wang J
    Kim K
    Friedman G
    Wang X
    Taupin V
    Crooks GM
    Kohn DB
    Gregory PD
    Holmes MC
    Cannon PM
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    Źródło:
    Nature biotechnology [Nat Biotechnol] 2010 Aug; Vol. 28 (8), pp. 839-47. Date of Electronic Publication: 2010 Jul 02.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Endonucleases/*genetics
    Genetic Engineering/*methods
    HIV Infections/*therapy
    Hematopoietic Stem Cell Transplantation/*methods
    Receptors, CCR5/*genetics
    Zinc Fingers/*genetics
    Animals ; Endonucleases/metabolism ; Gene Deletion ; HIV Infections/immunology ; HIV Infections/virology ; HIV-1/immunology ; HIV-1/metabolism ; Hematopoietic Stem Cells/metabolism ; Humans ; Mice ; Mice, Inbred NOD ; Mice, SCID ; Receptors, CCR5/metabolism ; Stem Cells/metabolism ; T-Lymphocytes/immunology ; T-Lymphocytes/metabolism ; Zinc Fingers/physiology
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency.
    Autorzy:
    Gruber TA; Divisions of Hematology and Oncology, Children's Hospital Los Angeles, 4650 Sunset Blvd., Los Angeles, CA 90027, USA.
    Shah AJ
    Hernandez M
    Crooks GM
    Abdel-Azim H
    Gupta S
    McKnight S
    White D
    Kapoor N
    Kohn DB
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    Źródło:
    Pediatric transplantation [Pediatr Transplant] 2009 Mar; Vol. 13 (2), pp. 244-50. Date of Electronic Publication: 2008 Sep 05.
    Typ publikacji:
    Case Reports; Journal Article
    MeSH Terms:
    Immunologic Deficiency Syndromes/*genetics
    Immunologic Deficiency Syndromes/*immunology
    Severe Combined Immunodeficiency/*genetics
    Severe Combined Immunodeficiency/*immunology
    Cytokines/genetics ; Exanthema/genetics ; Female ; Homeodomain Proteins/genetics ; Humans ; Immunologic Deficiency Syndromes/complications ; Infant ; Male ; Models, Genetic ; Mutation ; Phenotype ; Severe Combined Immunodeficiency/complications ; T-Lymphocytes/metabolism ; Time Factors ; Treatment Outcome ; VDJ Recombinases/genetics
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Genetic therapies against HIV.
    Autorzy:
    Rossi JJ; Division of Molecular Biology, Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, Duarte, California 91010, USA. />June CH
    Kohn DB
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    Źródło:
    Nature biotechnology [Nat Biotechnol] 2007 Dec; Vol. 25 (12), pp. 1444-54.
    Typ publikacji:
    Journal Article; Review
    MeSH Terms:
    Drug Design*
    Drug Delivery Systems/*methods
    Gene Targeting/*methods
    Genetic Therapy/*methods
    HIV Infections/*drug therapy
    HIV Infections/*genetics
    Vaccines, DNA/*therapeutic use
    HIV Infections/virology ; Humans
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia.
    Autorzy:
    Gruber TA; Division of Research Immunology/Bone Marrow Transplantation, Childrens Hospital Los Angeles 90027, USA.
    Skelton DC
    Kohn DB
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    Źródło:
    Cancer gene therapy [Cancer Gene Ther] 2005 Oct; Vol. 12 (10), pp. 818-24.
    Typ publikacji:
    Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Philadelphia Chromosome*
    Immunotherapy/*methods
    Interleukin-12/*therapeutic use
    Precursor Cell Lymphoblastic Leukemia-Lymphoma/*immunology
    Precursor Cell Lymphoblastic Leukemia-Lymphoma/*therapy
    Recombinant Proteins/*therapeutic use
    Animals ; Cell Line ; Immunologic Factors/genetics ; Immunologic Memory/immunology ; Interleukin-12/metabolism ; Male ; Mice ; Mice, Inbred BALB C ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/genetics ; Recombinant Proteins/metabolism ; T-Lymphocytes/immunology ; Vaccination/methods
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Occurrence of leukaemia following gene therapy of X-linked SCID.
    Autorzy:
    Kohn DB; Division of Research Immunology/BMT, Childrens Hospital Los Angeles, USC Keck School of Medicine, 4650 Sunset Boulevard, Los Angeles, California 90027, USA. />Sadelain M
    Glorioso JC
    Pokaż więcej
    Źródło:
    Nature reviews. Cancer [Nat Rev Cancer] 2003 Jul; Vol. 3 (7), pp. 477-88.
    Typ publikacji:
    Journal Article; Review
    MeSH Terms:
    Genetic Diseases, X-Linked/*therapy
    Genetic Therapy/*adverse effects
    Genetic Therapy/*methods
    Leukemia/*etiology
    Severe Combined Immunodeficiency/*therapy
    Cell Transformation, Neoplastic ; Clinical Trials as Topic ; Genetic Diseases, X-Linked/complications ; Genetic Diseases, X-Linked/genetics ; Genetic Vectors/genetics ; Humans ; Severe Combined Immunodeficiency/complications ; Severe Combined Immunodeficiency/genetics
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates.
    Autorzy:
    Schmidt M; Department I of Internal Medicine, University of Freiburg, Freiburg, Germany.
    Carbonaro DA
    Speckmann C
    Wissler M
    Bohnsack J
    Elder M
    Aronow BJ
    Nolta JA
    Kohn DB
    von Kalle C
    Pokaż więcej
    Źródło:
    Nature medicine [Nat Med] 2003 Apr; Vol. 9 (4), pp. 463-8. Date of Electronic Publication: 2003 Mar 17.
    Typ publikacji:
    Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.
    MeSH Terms:
    Antigens, CD34*
    Fetal Blood*
    Gene Transfer Techniques*
    Transduction, Genetic*
    Adenosine Deaminase/*genetics
    Severe Combined Immunodeficiency/*therapy
    Clone Cells ; Genetic Vectors ; Humans ; Infant, Newborn ; Polymerase Chain Reaction ; Retroviridae/genetics ; T-Lymphocytes/metabolism
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
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