id,collection,dc.contributor.author,dc.date.accessioned,dc.date.available,dc.date.issued,dc.description.abstract[en],dc.format.extent,dc.identifier.uri,dc.language,dc.rights.uri,dc.subject.ddc,dc.title,dc.type,dcterms.accessRights.openaire,dcterms.bibliographicCitation,dcterms.bibliographicCitation.doi,dcterms.bibliographicCitation.url,refubium.affiliation[de],refubium.mycore.derivateId,refubium.mycore.fudocsId,refubium.resourceType.isindependentpub "0ba438a5-560b-42cf-92ec-62aa8572b69f","fub188/15","Gundry, Michael C.||Brunetti, Lorenzo||Lin, Angelique||Mayle, Allison E.||Kitano, Ayumi||Wagner, Dimitrios||Hsu, Joanne I.||Hoegenauer, Kevin A.||Rooney, Cliona M.||Goodell, Margaret A.||Nakada, Daisuke","2018-06-08T10:25:39Z","2016-12-13T13:33:59.179Z","2016","Summary Our understanding of the mechanisms that regulate hematopoietic stem/progenitor cells (HSPCs) has been advanced by the ability to genetically manipulate mice; however, germline modification is time consuming and expensive. Here, we describe fast, efficient, and cost-effective methods to directly modify the genomes of mouse and human HSPCs using the CRISPR/Cas9 system. Using plasmid and virus-free delivery of guide RNAs alone into Cas9-expressing HSPCs or Cas9-guide RNA ribonucleoprotein (RNP) complexes into wild-type cells, we have achieved extremely efficient gene disruption in primary HSPCs from mouse (>60%) and human (∼75%). These techniques enabled rapid evaluation of the functional effects of gene loss of Eed, Suz12, and DNMT3A. We also achieved homology-directed repair in primary human HSPCs (>20%). These methods will significantly expand applications for CRISPR/Cas9 technologies for studying normal and malignant hematopoiesis.","10 S.","https://refubium.fu-berlin.de/handle/fub188/20403||http://dx.doi.org/10.17169/refubium-23706","eng","http://creativecommons.org/licenses/by-nc-nd/4.0/","600 Technik, Medizin, angewandte Wissenschaften::610 Medizin und Gesundheit","Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9","Wissenschaftlicher Artikel","open access","Cell Reports. - 17 (2016), 5, S. 1453–1461","10.1016/j.celrep.2016.09.092","http://dx.doi.org/10.1016/j.celrep.2016.09.092","Charité - Universitätsmedizin Berlin","FUDOCS_derivate_000000007436","FUDOCS_document_000000026032","no"