TY - GEN AU - Dever,Daniel P AU - Bak,Rasmus O AU - Reinisch,Andreas AU - Camarena,Joab AU - Washington,Gabriel AU - Nicolas,Carmencita E AU - Pavel-Dinu,Mara AU - Saxena,Nivi AU - Wilkens,Alec B AU - Mantri,Sruthi AU - Uchida,Nobuko AU - Hendel,Ayal AU - Narla,Anupama AU - Majeti,Ravindra AU - Weinberg,Kenneth I AU - Porteus,Matthew H TI - CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells SN - 1476-4687 PY - 2017///0321 KW - Alleles KW - Anemia, Sickle Cell KW - genetics KW - Animals KW - Antigens, CD34 KW - metabolism KW - CRISPR-Associated Proteins KW - CRISPR-Cas Systems KW - Cell Differentiation KW - Cell Lineage KW - Cell Separation KW - Dependovirus KW - Erythrocytes KW - Female KW - Flow Cytometry KW - Gene Editing KW - methods KW - Gene Targeting KW - Genes, Reporter KW - Genetic Therapy KW - Hematopoietic Stem Cells KW - Homologous Recombination KW - Humans KW - Magnets KW - Mice, Inbred NOD KW - Mice, SCID KW - Microspheres KW - Mutation KW - RNA, Messenger KW - biosynthesis KW - beta-Globins KW - beta-Thalassemia N1 - Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't UR - https://doi.org/10.1038/nature20134 ER -