المؤلفون: Kuo CY; Division of Allergy & Immunology, Department of Pediatrics, David Geffen School of Medicine at the University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address: ckuo@mednet.ucla.edu., Long JD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Campo-Fernandez B; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., de Oliveira S; Division of Hematology & Oncology, Department of Pediatrics, David Geffen School of Medicine at the University of California, Los Angeles, Los Angeles, CA 90095, USA., Cooper AR; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Romero Z; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Hoban MD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Joglekar AV; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Lill GR; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Kaufman ML; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Fitz-Gibbon S; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, Los Angeles, CA 90095, USA., Wang X; Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, CA 90095, USA., Hollis RP; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA; Division of Hematology & Oncology, Department of Pediatrics, David Geffen School of Medicine at the University of California, Los Angeles, Los Angeles, CA 90095, USA.

المصدر: Cell reports [Cell Rep] 2018 May 29; Vol. 23 (9), pp. 2606-2616.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101573691 Publication Model: Print Cited Medium: Internet ISSN: 2211-1247 (Electronic) NLM ISO Abbreviation: Cell Rep Subsets: MEDLINE

مواضيع طبية MeSH: Gene Editing*, Genetic Diseases, X-Linked/*genetics , Hematopoietic Stem Cells/*metabolism , Hyper-IgM Immunodeficiency Syndrome/*genetics, Animals ; Antigens, CD34/metabolism ; Base Sequence ; CD40 Ligand/metabolism ; CRISPR-Associated Protein 9/metabolism ; CRISPR-Cas Systems/genetics ; Cell Differentiation ; Cell Line ; Colony-Forming Units Assay ; DNA Repair ; DNA, Complementary/genetics ; Humans ; Mice ; T-Lymphocytes/metabolism ; Transcription Activator-Like Effector Nucleases/metabolism

المؤلفون: Cooper AR; Department of Microbiology, Immunology and Molecular Genetics and.; Molecular Biology Interdepartmental PhD Program, University of California, Los Angeles, Los Angeles, CA., Lill GR; Department of Microbiology, Immunology and Molecular Genetics and., Shaw K; Department of Microbiology, Immunology and Molecular Genetics and., Carbonaro-Sarracino DA; Department of Microbiology, Immunology and Molecular Genetics and., Davila A; Department of Microbiology, Immunology and Molecular Genetics and., Sokolic R; Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD; and., Candotti F; Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD; and., Pellegrini M; Department of Molecular, Cellular and Developmental Biology., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics and.; Molecular Biology Institute, and.; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA.

المصدر: Blood [Blood] 2017 May 11; Vol. 129 (19), pp. 2624-2635. Date of Electronic Publication: 2017 Mar 28.

نوع المنشور: Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural

بيانات الدورية: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE

مواضيع طبية MeSH: Adenosine Deaminase/*deficiency , Agammaglobulinemia/*genetics , Agammaglobulinemia/*therapy , Antineoplastic Agents, Alkylating/*therapeutic use , Busulfan/*therapeutic use , Gammaretrovirus/*genetics , Genetic Therapy/*methods , Genetic Vectors/*therapeutic use , Severe Combined Immunodeficiency/*genetics , Severe Combined Immunodeficiency/*therapy, Adaptor Proteins, Signal Transducing/genetics ; Adenosine Deaminase/genetics ; Agammaglobulinemia/pathology ; Child ; DNA-Binding Proteins/genetics ; Genetic Vectors/genetics ; Humans ; LIM Domain Proteins/genetics ; MDS1 and EVI1 Complex Locus Protein ; Proto-Oncogene Proteins/genetics ; Proto-Oncogenes/genetics ; Severe Combined Immunodeficiency/pathology ; T-Lymphocytes/cytology ; T-Lymphocytes/drug effects ; T-Lymphocytes/metabolism ; T-Lymphocytes/pathology ; Transcription Factors/genetics

SCR Disease Name: Severe combined immunodeficiency due to adenosine deaminase deficiency

المؤلفون: Hoban MD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Lumaquin D; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Kuo CY; Division of Allergy and Immunology, Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, California, USA., Romero Z; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Long J; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA.; Biology Department, California State University, Northridge, California, USA., Ho M; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Young CS; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, California, USA.; Molecular Biology Interdepartmental PhD Program (MBIDP), University of California, Los Angeles, California, USA., Mojadidi M; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Fitz-Gibbon S; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, California, USA.; Institute for Genomics and Proteomics, University of California, Los Angeles, California, USA., Cooper AR; Molecular Biology Interdepartmental PhD Program (MBIDP), University of California, Los Angeles, California, USA., Lill GR; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Urbinati F; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Campo-Fernandez B; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Bjurstrom CF; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Pellegrini M; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, California, USA.; Institute for Genomics and Proteomics, University of California, Los Angeles, California, USA., Hollis RP; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA.; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, California, USA.

المصدر: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2016 Sep; Vol. 24 (9), pp. 1561-9. Date of Electronic Publication: 2016 Jul 29.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

مواضيع طبية MeSH: CRISPR-Cas Systems* , Gene Editing* , Mutation* , Targeted Gene Repair*, Anemia, Sickle Cell/*genetics , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/therapy ; Base Sequence ; Cell Line ; DNA Cleavage ; Gene Targeting ; Genetic Loci ; Humans ; Protein Binding ; RNA, Guide, CRISPR-Cas Systems ; Transcription Activator-Like Effector Nucleases/metabolism

المؤلفون: Chin CJ; Department of Pathology & Laboratory Medicine, David Geffen School of Medicine (DGSOM)., Cooper AR; Molecular Biology Interdepartmental PhD Program, DGSOM University of California Los Angeles.; Department of Microbiology, Immunology and Molecular Genetics, DGSOM, DGSOM University of California Los Angeles., Lill GR; Department of Microbiology, Immunology and Molecular Genetics, DGSOM, DGSOM University of California Los Angeles., Evseenko D; Department of Orthopedic Surgery, Keck School of Medicine of University of Southern California (USC). All in, Los Angeles, CA, United States., Zhu Y; Department of Pathology & Laboratory Medicine, David Geffen School of Medicine (DGSOM)., He CB; Department of Pathology & Laboratory Medicine, David Geffen School of Medicine (DGSOM)., Casero D; Department of Pathology & Laboratory Medicine, David Geffen School of Medicine (DGSOM)., Pellegrini M; Department of Molecular, Cell and Development Biology, DGSOM University of California Los Angeles.; Molecular Biology Institute (MBI)., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, DGSOM, DGSOM University of California Los Angeles.; Molecular Biology Institute (MBI).; Department of Pediatrics, DGSOM University of California Los Angeles.; Broad Stem Cell Research Center (BSCRC), DGSOM University of California Los Angeles.; Jonsson Comprehensive Cancer Center (JCCC), DGSOM University of California Los Angeles., Crooks GM; Department of Pathology & Laboratory Medicine, David Geffen School of Medicine (DGSOM).; Department of Pediatrics, DGSOM University of California Los Angeles.; Broad Stem Cell Research Center (BSCRC), DGSOM University of California Los Angeles.; Department of Pathology & Laboratory Medicine, DGSOM University of California Los Angeles.

المصدر: Stem cells (Dayton, Ohio) [Stem Cells] 2016 May; Vol. 34 (5), pp. 1239-50. Date of Electronic Publication: 2016 Mar 28.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Oxford University Press Country of Publication: England NLM ID: 9304532 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1549-4918 (Electronic) Linking ISSN: 10665099 NLM ISO Abbreviation: Stem Cells Subsets: MEDLINE

مواضيع طبية MeSH: Cell Differentiation* , Genetic Techniques*, Mesoderm/*cytology , Multipotent Stem Cells/*cytology, Animals ; Antigens, CD/metabolism ; Cell Line ; Cell Lineage ; Cell Separation ; Clone Cells ; Flow Cytometry ; Humans ; Lentivirus/metabolism ; Mice

المؤلفون: Bjurström CF; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Mojadidi M; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Phillips J; Howard Hughes Medical Institute, University of California, Los Angeles, Los Angeles, California, USA., Kuo C; Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA., Lai S; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Lill GR; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Cooper A; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Kaufman M; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Urbinati F; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Wang X; Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, California, USA., Hollis RP; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Kohn DB; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA; Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA. Electronic address: dkohn@mednet.ucla.edu.

المصدر: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2016; Vol. 5, pp. e351.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: Print Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE

المؤلفون: Hoban MD; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Cost GJ; Sangamo BioSciences Inc., Richmond, CA;, Mendel MC; Sangamo BioSciences Inc., Richmond, CA;, Romero Z; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Kaufman ML; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Joglekar AV; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Ho M; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Lumaquin D; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Gray D; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Lill GR; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Cooper AR; Molecular Biology Interdepartmental PhD Program, University of California, Los Angeles, Los Angeles, CA;, Urbinati F; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Senadheera S; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Zhu A; Sangamo BioSciences Inc., Richmond, CA;, Liu PQ; Sangamo BioSciences Inc., Richmond, CA;, Paschon DE; Sangamo BioSciences Inc., Richmond, CA;, Zhang L; Sangamo BioSciences Inc., Richmond, CA;, Rebar EJ; Sangamo BioSciences Inc., Richmond, CA;, Wilber A; Department of Medical Microbiology, Immunology and Cell Biology, Southern Illinois University School of Medicine, Springfield, IL;, Wang X; Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, CA; and., Gregory PD; Sangamo BioSciences Inc., Richmond, CA;, Holmes MC; Sangamo BioSciences Inc., Richmond, CA;, Reik A; Sangamo BioSciences Inc., Richmond, CA;, Hollis RP; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA.

المصدر: Blood [Blood] 2015 Apr 23; Vol. 125 (17), pp. 2597-604. Date of Electronic Publication: 2015 Mar 02.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy* , Mutation*, Anemia, Sickle Cell/*genetics , Anemia, Sickle Cell/*therapy , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/pathology ; Animals ; Antigens, CD34/analysis ; Base Sequence ; Bone Marrow Cells/metabolism ; Bone Marrow Cells/pathology ; Cells, Cultured ; Endodeoxyribonucleases/metabolism ; Fetal Blood/transplantation ; Genetic Loci ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/pathology ; Humans ; Mice ; Mice, Inbred NOD ; Mice, SCID ; Molecular Sequence Data ; Zinc Fingers

المؤلفون: Cooper AR; Molecular Biology Interdepartmental PhD Program, University of California, Los Angeles, Los Angeles, CA 90095, USA., Lill GR; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Gschweng EH; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA 90095, USA Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA dkohn1@mednet.ucla.edu.

المصدر: Nucleic acids research [Nucleic Acids Res] 2015 Jan; Vol. 43 (1), pp. 682-90. Date of Electronic Publication: 2014 Dec 17.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Vectors* , Introns* , Promoter Regions, Genetic*, Lentivirus/*genetics , Ubiquitin C/*genetics, Enhancer Elements, Genetic ; Exons ; Gene Expression ; HEK293 Cells ; Humans ; K562 Cells ; Peptide Elongation Factor 1/genetics ; RNA Splicing