المؤلفون: Wilkes, MC, Siva, K, Chen, J, Varetti, G, Youn, MY, Chae, H, Ek, F, Olsson, R, Lundback, T, Dever, DP, Nishimura, T, Narla, A, Glader, B, Nakauchi, H, Porteus, MH, Repellin, CE, Gazda, HT, Lin, S, Serrano, M, Flygare, J, Sakamoto, KM

المصدر: Nature communications. 11(1):3344

مصطلحات موضوعية: Medicin och hälsovetenskap, Klinisk medicin, Hematologi, Medicinska och farmaceutiska grundvetenskaper, Cell- och molekylärbiologi

URL الوصول: http://kipublications.ki.se/Default.aspx?queryparsed=id:144133480

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المؤلفون: Perez-Bermejo JA; Graphite Bio, South San Francisco, CA, USA., Efagene O; Graphite Bio, South San Francisco, CA, USA., Matern WM; Graphite Bio, South San Francisco, CA, USA., Holden JK; Graphite Bio, South San Francisco, CA, USA., Kabir S; Graphite Bio, South San Francisco, CA, USA., Chew GM; Graphite Bio, South San Francisco, CA, USA., Andreoletti G; Graphite Bio, South San Francisco, CA, USA., Catton E; Graphite Bio, South San Francisco, CA, USA., Ennis CL; Graphite Bio, South San Francisco, CA, USA., Garcia A; Graphite Bio, South San Francisco, CA, USA., Gerstenberg TL; Graphite Bio, South San Francisco, CA, USA., Hill KA; Graphite Bio, South San Francisco, CA, USA., Jain A; Graphite Bio, South San Francisco, CA, USA., Krassovsky K; Graphite Bio, South San Francisco, CA, USA., Lalisan CD; Graphite Bio, South San Francisco, CA, USA., Lord D; Graphite Bio, South San Francisco, CA, USA., Quejarro BJ; Graphite Bio, South San Francisco, CA, USA., Sales-Lee J; Graphite Bio, South San Francisco, CA, USA., Shah M; Graphite Bio, South San Francisco, CA, USA., Silva BJ; Graphite Bio, South San Francisco, CA, USA., Skowronski J; Graphite Bio, South San Francisco, CA, USA., Strukov YG; Graphite Bio, South San Francisco, CA, USA., Thomas J; Graphite Bio, South San Francisco, CA, USA., Veraz M; Graphite Bio, South San Francisco, CA, USA., Vijay T; Graphite Bio, South San Francisco, CA, USA., Wallace KA; Graphite Bio, South San Francisco, CA, USA., Yuan Y; Graphite Bio, South San Francisco, CA, USA., Grogan JL; Graphite Bio, South San Francisco, CA, USA., Wienert B; Graphite Bio, South San Francisco, CA, USA., Lahiri P; Graphite Bio, South San Francisco, CA, USA., Treusch S; Graphite Bio, South San Francisco, CA, USA., Dever DP; Graphite Bio, South San Francisco, CA, USA., Soros VB; Graphite Bio, South San Francisco, CA, USA., Partridge JR; Graphite Bio, South San Francisco, CA, USA., Seim KL; Graphite Bio, South San Francisco, CA, USA. scicomms@graphitebio.com.

المصدر: Nature communications [Nat Commun] 2024 Mar 23; Vol. 15 (1), pp. 2625. Date of Electronic Publication: 2024 Mar 23.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

مواضيع طبية MeSH: CRISPR-Cas Systems* , Recombinational DNA Repair*, Humans ; Gene Editing/methods ; DNA Repair ; DNA End-Joining Repair

المؤلفون: Baik R; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.; Molecular Biology Program, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Cromer MK; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Glenn SE; Integrated DNA Technologies, Inc., Coralville, IA, USA., Vakulskas CA; Integrated DNA Technologies, Inc., Coralville, IA, USA., Chmielewski KO; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, 79106, Freiburg, Germany.; Center for Chronic Immunodeficiency, University of Freiburg, 79106, Freiburg, Germany.; Ph.D. Program, Faculty of Biology, University of Freiburg, 79104, Freiburg, Germany., Dudek AM; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Feist WN; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Klermund J; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, 79106, Freiburg, Germany.; Center for Chronic Immunodeficiency, University of Freiburg, 79106, Freiburg, Germany., Shipp S; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Cathomen T; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, 79106, Freiburg, Germany.; Center for Chronic Immunodeficiency, University of Freiburg, 79106, Freiburg, Germany., Dever DP; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Porteus MH; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA. mporteus@stanford.edu.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA. mporteus@stanford.edu.

المصدر: Nature communications [Nat Commun] 2024 Jan 02; Vol. 15 (1), pp. 111. Date of Electronic Publication: 2024 Jan 02.

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

بيانات الدورية: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

مواضيع طبية MeSH: Gene Editing*/methods , Hematopoietic Stem Cell Transplantation*, Humans ; Animals ; Mice ; Hematopoietic Stem Cells/metabolism ; Recombinant Proteins/metabolism ; Peptides/metabolism ; CRISPR-Cas Systems

المؤلفون: Lattanzi A; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.; Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA., Camarena J; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Lahiri P; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA., Segal H; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA., Srifa W; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Vakulskas CA; Integrated DNA Technologies Inc., Coralville, IA 52241, USA., Frock RL; Department of Radiation Oncology, Stanford University, Stanford, CA 94305, USA., Kenrick J; Department of Radiation Oncology, Stanford University, Stanford, CA 94305, USA., Lee C; APC Microbiome Ireland, University College Cork, T12 YN60 Cork, Ireland., Talbott N; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA., Skowronski J; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA., Cromer MK; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Charlesworth CT; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Bak RO; Department of Biomedicine, Aarhus University, DK-8000 Aarhus, Denmark.; Aarhus Institute of Advanced Studies (AIAS), Aarhus University, DK-8000 Aarhus, Denmark., Mantri S; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Bao G; Department of Bioengineering, Rice University, Houston, TX 77006, USA., DiGiusto D; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA., Tisdale J; Molecular and Clinical Hematology Branch, NHLBI, Bethesda, MD 20814, USA., Wright JF; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.; Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA., Bhatia N; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.; Deceased., Roncarolo MG; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.; Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA.; Institute of Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA 94305, USA., Dever DP; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. dpdever@gmail.com mporteus@stanford.edu., Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. dpdever@gmail.com mporteus@stanford.edu.; Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA.; Institute of Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA 94305, USA.

المصدر: Science translational medicine [Sci Transl Med] 2021 Jun 16; Vol. 13 (598).

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

بيانات الدورية: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE

مواضيع طبية MeSH: Anemia, Sickle Cell*/genetics , Anemia, Sickle Cell*/therapy , Heterocyclic Compounds*, Animals ; CRISPR-Cas Systems/genetics ; Gene Editing ; Hematopoietic Stem Cell Mobilization ; Hematopoietic Stem Cells ; Humans ; Mice ; Reproducibility of Results ; beta-Globins/genetics

المؤلفون: Fañanas-Baquero S; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Quintana-Bustamante O; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Dever DP; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Alberquilla O; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Sanchez-Dominguez R; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Camarena J; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Ojeda-Perez I; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Dessy-Rodriguez M; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Turk R; Integrated DNA Technologies, Coralville, IA 52241, USA., Schubert MS; Integrated DNA Technologies, Coralville, IA 52241, USA., Lattanzi A; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Xu L; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Lopez-Lorenzo JL; Hospital Universitario Fundación Jiménez Díaz, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD, UAM), Madrid 28015, Spain., Bianchi P; UOC Ematologia, Fisiopatologia delle Anemie, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano, Milan 20122, Italy., Bueren JA; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain., Behlke MA; Integrated DNA Technologies, Coralville, IA 52241, USA., Porteus M; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Segovia JC; Biomedical Innovation Unit, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid 28040, Spain.; Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez (IIS-FJD, UAM), Madrid 28040, Spain.

المصدر: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2021 May 14; Vol. 22, pp. 237-248. Date of Electronic Publication: 2021 May 14 (Print Publication: 2021).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

المؤلفون: Cromer MK; Department of Pediatrics, Stanford University, Stanford, CA, USA., Camarena J; Department of Pediatrics, Stanford University, Stanford, CA, USA., Martin RM; Department of Pediatrics, Stanford University, Stanford, CA, USA., Lesch BJ; Department of Pediatrics, Stanford University, Stanford, CA, USA., Vakulskas CA; Integrated DNA Technologies, Inc., Coralville, IA, USA., Bode NM; Integrated DNA Technologies, Inc., Coralville, IA, USA., Kurgan G; Integrated DNA Technologies, Inc., Coralville, IA, USA., Collingwood MA; Integrated DNA Technologies, Inc., Coralville, IA, USA., Rettig GR; Integrated DNA Technologies, Inc., Coralville, IA, USA., Behlke MA; Integrated DNA Technologies, Inc., Coralville, IA, USA., Lemgart VT; Department of Pediatrics, Stanford University, Stanford, CA, USA., Zhang Y; Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX, USA., Goyal A; Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX, USA., Zhao F; Department of Medicine, Division of Hematology, Stanford University, Stanford, CA, USA.; Institute of Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, USA., Ponce E; Department of Pediatrics, Stanford University, Stanford, CA, USA., Srifa W; Department of Pediatrics, Stanford University, Stanford, CA, USA., Bak RO; Department of Biomedicine, Aarhus University, Aarhus, Denmark.; Aarhus Institute of Advanced Studies, Aarhus University, Aarhus, Denmark., Uchida N; Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA., Majeti R; Department of Medicine, Division of Hematology, Stanford University, Stanford, CA, USA.; Institute of Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, USA., Sheehan VA; Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX, USA., Tisdale JF; Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA., Dever DP; Department of Pediatrics, Stanford University, Stanford, CA, USA. ddever1@stanford.edu., Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA, USA. mporteus@stanford.edu.; Institute of Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, USA. mporteus@stanford.edu.

المصدر: Nature medicine [Nat Med] 2021 Apr; Vol. 27 (4), pp. 677-687. Date of Electronic Publication: 2021 Mar 18.

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

بيانات الدورية: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy*, Hematopoietic Stem Cells/*metabolism , Hemoglobins/*metabolism , alpha-Globins/*genetics , beta-Globins/*genetics , beta-Thalassemia/*genetics , beta-Thalassemia/*therapy, Anemia, Sickle Cell/pathology ; Animals ; Antigens, CD34/metabolism ; Dependovirus/genetics ; Erythrocytes/metabolism ; Gene Editing ; Genes, Reporter ; Genetic Loci ; Hematopoietic Stem Cell Transplantation ; Humans ; Mice ; Promoter Regions, Genetic/genetics

المؤلفون: Wilkinson AC; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA. adamcw@stanford.edu.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA. adamcw@stanford.edu., Dever DP; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Baik R; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Camarena J; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA., Hsu I; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Charlesworth CT; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Morita C; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA., Nakauchi H; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA. nakauchi@stanford.edu.; Department of Genetics, Stanford University School of Medicine, Stanford, CA, USA. nakauchi@stanford.edu.; Division of Stem Cell Therapy, Distinguished Professor Unit, The Institute of Medical Science, The University of Tokyo, Tokyo, 108-8639, Japan. nakauchi@stanford.edu., Porteus MH; Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Lorry I. Lokey Stem Cell Research Building, 265 Campus Drive, Stanford, CA, USA. mporteus@stanford.edu.; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA. mporteus@stanford.edu.

المصدر: Nature communications [Nat Commun] 2021 Jan 29; Vol. 12 (1), pp. 686. Date of Electronic Publication: 2021 Jan 29.

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: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

مواضيع طبية MeSH: Anemia, Sickle Cell/*therapy , Erythropoiesis/*genetics , Genetic Therapy/*methods , Hematopoietic Stem Cell Transplantation/*methods , beta-Globins/*genetics, Anemia, Sickle Cell/blood ; Anemia, Sickle Cell/diagnosis ; Anemia, Sickle Cell/genetics ; Animals ; CRISPR-Cas Systems/genetics ; Combined Modality Therapy/methods ; Dependovirus ; Disease Models, Animal ; Female ; Gene Editing/methods ; Gene Knock-In Techniques ; Genetic Vectors/genetics ; Humans ; Mice ; Mice, Transgenic ; Parvovirinae/genetics ; Transplantation, Autologous/methods

SCR Organism: Adeno-associated virus-1

المؤلفون: Sharma R; Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, 94305, USA., Dever DP; Department of Pediatrics, Stanford University, Stanford, CA, 94305, USA., Lee CM; Department of Bioengineering, Rice University, Houston, TX, 77030, USA., Azizi A; Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, 94305, USA., Pan Y; Department of Bioengineering, Rice University, Houston, TX, 77030, USA., Camarena J; Department of Pediatrics, Stanford University, Stanford, CA, 94305, USA., Köhnke T; Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, 94305, USA., Bao G; Department of Bioengineering, Rice University, Houston, TX, 77030, USA. gb20@rice.edu., Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA, 94305, USA. mporteus@stanford.edu., Majeti R; Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, 94305, USA. rmajeti@stanford.edu.

المصدر: Nature communications [Nat Commun] 2021 Jan 20; Vol. 12 (1), pp. 472. Date of Electronic Publication: 2021 Jan 20.

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: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

مواضيع طبية MeSH: Alleles* , Clone Cells* , Hematopoietic Stem Cells* , Recombination, Genetic*, Gene Targeting/*methods, Animals ; CRISPR-Cas Systems ; Clustered Regularly Interspaced Short Palindromic Repeats ; Female ; Gene Editing/methods ; Genetic Therapy/methods ; Humans ; Mice ; Mutation ; Targeted Gene Repair/methods

المؤلفون: Park SH; Department of Bioengineering, Rice University, Houston, TX 77030, USA., Lee CM; Department of Bioengineering, Rice University, Houston, TX 77030, USA., Dever DP; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Davis TH; Department of Bioengineering, Rice University, Houston, TX 77030, USA., Camarena J; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Srifa W; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Zhang Y; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA., Paikari A; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA., Chang AK; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA., Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA., Sheehan VA; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA., Bao G; Department of Bioengineering, Rice University, Houston, TX 77030, USA.

المصدر: Nucleic acids research [Nucleic Acids Res] 2019 Sep 05; Vol. 47 (15), pp. 7955-7972.

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 Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE

مواضيع طبية MeSH: Anemia, Sickle Cell/*therapy , Gene Editing/*methods , Hematopoietic Stem Cell Transplantation/*methods , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/genetics ; Animals ; CRISPR-Cas Systems ; Cell Line, Tumor ; Cells, Cultured ; Erythrocytes/metabolism ; Genetic Therapy/methods ; Humans ; K562 Cells ; Mice ; Mice, Inbred NOD ; Mice, Knockout ; Mice, SCID ; Treatment Outcome