In vivo genome editing restores haemostasis in a mouse model of haemophilia

Nature

Volumn 475, Issue 7355, 2011, Pages 217-221

  Li, Hojun ^a   Haurigot, Virginia ^a   Doyon, Yannick ^b   Li, Tianjian ^b   Wong, Sunnie Y ^b   Bhagwat, Anand S ^a   Malani, Nirav ^c   Anguela, Xavier M ^a   Sharma, Rajiv ^a   Ivanciu, Lacramiora ^a   Murphy, Samuel L ^a   Finn, Jonathan D ^a   Khazi, Fayaz R ^a   Zhou, Shangzhen ^a   Paschon, David E ^b   Rebar, Edward J ^b   Bushman, Frederic D ^c   Gregory, Philip D ^b   Holmes, Michael C ^b   High, Katherine A ^a,d  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b SANGAMO BIOSCIENCES INC   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

^d HOWARD HUGHES MEDICAL INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ZINC FINGER PROTEIN;

CYTOLOGY; GENOME; HEMATOLOGY; HOMOLOGY; IN SITU MEASUREMENT; MUTATION; RODENT;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD CLOTTING TIME; DOUBLE STRANDED DNA BREAK; EXON; GENE ACTIVITY; GENE INSERTION; GENE LOCUS; GENE TARGETING; GENETIC ANALYSIS; GENOME; GENOME EDITING; HEMOPHILIA B; HEMOSTASIS; HUMAN; HUMAN CELL; IN VIVO STUDY; LIVER REGENERATION; MOUSE; NONHUMAN; PRIORITY JOURNAL; SEQUENCE HOMOLOGY;

ANIMALS; BASE SEQUENCE; CELL LINE, TUMOR; DISEASE MODELS, ANIMAL; DNA BREAKS, DOUBLE-STRANDED; DNA REPAIR; ENDONUCLEASES; EXONS; FACTOR IX; GENE TARGETING; GENE THERAPY; GENETIC VECTORS; GENOME; HEK293 CELLS; HEMOPHILIA B; HEMOSTASIS; HUMANS; INTRONS; LIVER; LIVER REGENERATION; MICE; MICE, INBRED C57BL; MUTATION; PHENOTYPE; SEQUENCE HOMOLOGY; ZINC FINGERS;

EID: 79960424171     PISSN: 00280836     EISSN: 14764687     Source Type: Journal    
DOI: 10.1038/nature10177     Document Type: Article

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