المؤلفون: Mary Oakley Strasser, Mary Oakley Strasser, Michael Hipp, MD, Connor Hoban, MD, Michael Inadomi, MD, Colleen Macke, MD, Juan Noda, Kiki Ogu, MD, Balaji Pandian, Stewart Wang, MD, PhD

المصدر: Michigan Journal of Medicine: vol. 4, no. 2

مصطلحات الفهرس: text

URL: http://hdl.handle.net/2027/spo.13761231.0004.205

المؤلفون: Romero Z; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Lomova A; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, Los Angeles, CA, USA., Said S; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Miggelbrink A; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Kuo CY; Division of Allergy & Immunology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA., Campo-Fernandez B; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Hoban MD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Masiuk KE; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Clark DN; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Long J; Division of Allergy & Immunology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA., Sanchez JM; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Velez M; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Miyahira E; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Zhang R; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Brown D; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Wang X; Department of Medicine Statistics Core, University of California, Los Angeles, Los Angeles, CA, USA., Kurmangaliyev YZ; Department of Biological Chemistry, HHMI, University of California, Los Angeles, Los Angeles, CA, USA., Hollis RP; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, Los Angeles, CA, USA; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA. Electronic address: dkohn1@mednet.ucla.edu.

المصدر: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2019 Aug 07; Vol. 27 (8), pp. 1389-1406. Date of Electronic Publication: 2019 May 24.

نوع المنشور: Journal Article; 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: Gene Editing* , Mutation*, Anemia, Sickle Cell/*genetics , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/metabolism ; Anemia, Sickle Cell/therapy ; CRISPR-Cas Systems ; Dependovirus ; Endonucleases/genetics ; Gene Expression ; Gene Targeting ; Genetic Therapy ; Genetic Vectors/genetics ; Humans ; Parvovirinae/genetics ; Tissue Donors ; Transduction, Genetic ; Zinc Finger Nucleases/genetics

SCR Organism: Adeno-associated virus-1

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: Long J; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA; Biology Department, California State University, Northridge, Northridge, CA 91330, USA., Hoban MD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA., Cooper AR; Molecular Biology Interdepartmental Ph.D. Program, 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., Kuo CY; Division of Allergy and Immunology, Department of Pediatrics, David Geffen School of Medicine, 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., Lumaquin D; Department of Microbiology, Immunology and Molecular Genetics, 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., Wang X; Department of Internal Medicine and Health Services Research, 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; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, 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. Electronic address: zulemar@ucla.edu.

المصدر: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2018 Feb 07; Vol. 26 (2), pp. 468-479. Date of Electronic Publication: 2017 Nov 09.

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

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: Gene Editing* , Genetic Variation*, Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Gene Conversion ; Gene Rearrangement ; Gene Targeting ; High-Throughput Nucleotide Sequencing ; Humans ; Nucleic Acid Amplification Techniques ; Translocation, Genetic

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: Pinello L; Department of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.; Department of Biostatistics, Harvard TH Chan School of Public Health, Boston, Massachusetts, USA., Canver MC; Division of Hematology/Oncology, Boston Children's Hospital, Boston, Massachusetts, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.; Department of Pediatrics, Harvard Medical School and Harvard Stem Cell Institute, Harvard University, Boston, Massachusetts, USA., Hoban MD; Department of Microbiology, Immunology, and Molecular Genetics, Eli &Edythe Broad Center of Regenerative Medicine &Stem Cell Research, University of California, Los Angeles, Los Angeles, California, USA., Orkin SH; Division of Hematology/Oncology, Boston Children's Hospital, Boston, Massachusetts, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.; Department of Pediatrics, Harvard Medical School and Harvard Stem Cell Institute, Harvard University, Boston, Massachusetts, USA.; Howard Hughes Medical Institute, Boston, Massachusetts, USA., Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, Eli &Edythe Broad Center of Regenerative Medicine &Stem Cell Research, University of California, Los Angeles, Los Angeles, California, USA., Bauer DE; Division of Hematology/Oncology, Boston Children's Hospital, Boston, Massachusetts, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.; Department of Pediatrics, Harvard Medical School and Harvard Stem Cell Institute, Harvard University, Boston, Massachusetts, USA., Yuan GC; Department of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.; Department of Biostatistics, Harvard TH Chan School of Public Health, Boston, Massachusetts, USA.

المصدر: Nature biotechnology [Nat Biotechnol] 2016 Jul 12; Vol. 34 (7), pp. 695-7.

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

بيانات الدورية: Publisher: Nature America Publishing Country of Publication: United States NLM ID: 9604648 Publication Model: Print Cited Medium: Internet ISSN: 1546-1696 (Electronic) Linking ISSN: 10870156 NLM ISO Abbreviation: Nat Biotechnol Subsets: MEDLINE

مواضيع طبية MeSH: Models, Genetic* , Software*, CRISPR-Cas Systems/*genetics , Clustered Regularly Interspaced Short Palindromic Repeats/*genetics , Gene Editing/*methods , High-Throughput Nucleotide Sequencing/*methods , Sequence Analysis, DNA/*methods, Algorithms ; Animals ; Computer Simulation ; Humans

المؤلفون: Hoban MD; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA; and Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA., Bauer DE; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA; and Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA.

المصدر: Blood [Blood] 2016 May 26; Vol. 127 (21), pp. 2525-35. Date of Electronic Publication: 2016 Apr 06.

نوع المنشور: Journal Article; Review; 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: Gene Editing/*methods , Hematologic Diseases/*genetics , Hematologic Diseases/*therapy , Targeted Gene Repair/*methods, Animals ; Humans

المؤلفون: Hoban MD; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA; and., Orkin SH; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA; and Howard Hughes Medical Institute, Boston, MA., Bauer DE; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA; and.

المصدر: Blood [Blood] 2016 Feb 18; Vol. 127 (7), pp. 839-48. Date of Electronic Publication: 2016 Jan 12.

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

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: Anemia, Sickle Cell/*genetics , Anemia, Sickle Cell/*therapy , Genetic Therapy/*methods, Clinical Trials as Topic ; Gene Expression Regulation ; Genetic Vectors/genetics ; Genetic Vectors/therapeutic use ; Genome, Human ; Humans

المؤلفون: Hoban MD; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California., Romero Z; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California., Cost GJ; Sangamo BioSciences, Richmond, California., Mendel M; Sangamo BioSciences, Richmond, California., Holmes M; Sangamo BioSciences, Richmond, California., Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California.; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, California.

المصدر: Current protocols in stem cell biology [Curr Protoc Stem Cell Biol] 2016 Feb 03; Vol. 36, pp. 5B.4.1-5B.4.10. Date of Electronic Publication: 2016 Feb 03.

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

بيانات الدورية: Country of Publication: United States NLM ID: 101470226 Publication Model: Electronic Cited Medium: Internet ISSN: 1938-8969 (Electronic) Linking ISSN: 19388969 NLM ISO Abbreviation: Curr Protoc Stem Cell Biol Subsets: MEDLINE

مواضيع طبية MeSH: Genome, Human* , Hematopoietic Stem Cells*/cytology , Hematopoietic Stem Cells*/metabolism , Lentivirus*, Drug Delivery Systems/*methods , Electroporation/*methods , Transduction, Genetic/*methods, Animals ; Antigens, CD34 ; Humans

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