Efficient genome editing in hematopoietic stem cells with helper-dependent Ad5/35 vectors expressing site-specific endonucleases under microRNA regulation

Molecular Therapy Methods and Clinical Development

Volumn 2, Issue , 2015, Pages 14057-

  Saydaminova, Kamola ^a   Ye, Xun ^b   Wang, Hongjie ^a   Richter, Maximilian ^a   Ho, Martin ^a   Chen, HongZhuan ^b   Xu, Ning ^b   Kim, Jin Soo ^c   Papapetrou, Eirini ^a   Holmes, Michael C ^d   Gregory, Philip D ^d   Palmer, Donna ^e   Ng, Philip ^e   Ehrhardt, Anja ^f   Lieber, André ^a,g  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^b SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

^c Institute for Basic Science   (South Korea)

^d SANGAMO BIOSCIENCES INC   (United States)

^e BAYLOR COLLEGE OF MEDICINE   (United States)

^f UNIVERSITY OF WITTEN HERDECKE   (Germany)

^g UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENOMIC DNA; HELPER DEPENDENT ADENOVIRAL VECTOR; MESSENGER RNA; MICRORNA; MICRORNA 183; MICRORNA 218; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE; CHEMOKINE RECEPTOR CCR5; GLOBIN; MICRORNA 215; RESTRICTION ENDONUCLEASE;

ADULT; ALLELE; ARTICLE; CELL LINEAGE; CONTROLLED STUDY; ENGRAFTMENT; FLOW CYTOMETRY; GENE DISRUPTION; GENE EDITING; GENE EXPRESSION; GENE EXPRESSION PROFILING; GENE EXPRESSION SYSTEM; GENETIC REGULATION; GLOBIN GENE; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; INVERTED TERMINAL REPEAT; MOUSE; NONHUMAN; PERIPHERAL BLOOD MONONUCLEAR CELL; PRIORITY JOURNAL; PROMOTER REGION; REAL TIME POLYMERASE CHAIN REACTION; REGULATOR GENE; RESTRICTION MAPPING; SHUTTLE VECTOR; TRANSGENE; VIRUS PARTICLE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; CELL VIABILITY; COLONY FORMATION; DNA SEQUENCE; GENE EXPRESSION REGULATION; GENE INACTIVATION; PROTEIN EXPRESSION; TARGETED GENE REPAIR;

EID: 84928061468     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1038/mtm.2014.57     Document Type: Article

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