Targeted genome editing in human repopulating haematopoietic stem cells

Nature

Volumn 510, Issue 7504, 2014, Pages 235-240

  Genovese, Pietro ^a   Schiroli, Giulia ^a,b   Escobar, Giulia ^a,b   Di Tomaso, Tiziano ^a   Firrito, Claudia ^a   Calabria, Andrea ^a   Moi, Davide ^a,f   Mazzieri, Roberta ^a,f   Bonini, Chiara ^c   Holmes, Michael C ^d   Gregory, Philip D ^d   Van Der Burg, Mirjam ^e   Gentner, Bernhard ^a,b   Montini, Eugenio ^a   Lombardo, Angelo ^a,b   Naldini, Luigi ^a,b  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^c SAN RAFFAELE HOSPITAL   (Italy)

^d SANGAMO BIOSCIENCES INC   (United States)

^e ERASMUS MEDICAL CENTER   (Netherlands)

^f UNIVERSITY OF QUEENSLAND   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA;

CELL ORGANELLE; GENE EXPRESSION; GENETIC ANALYSIS; GENOME; GROWTH; MUTATION; RODENT;

ARTICLE; DNA REPAIR; GENE; GENE MUTATION; GENE TARGETING; GENE TRANSFER; GENETIC ENGINEERING AND GENE TECHNOLOGY; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; IL2RG GENE; LYMPHOID CELL; NONHUMAN; PRIORITY JOURNAL; TARGETED GENOME EDITING; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

MUS;

ANIMALS; ANTIGENS, CD34; DNA, COMPLEMENTARY; ENDONUCLEASES; FETAL BLOOD; GENE TARGETING; GENOME, HUMAN; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; INTERLEUKIN RECEPTOR COMMON GAMMA SUBUNIT; MALE; MICE; MUTATION; TARGETED GENE REPAIR; X-LINKED COMBINED IMMUNODEFICIENCY DISEASES;

EID: 84902315464     PISSN: 00280836     EISSN: 14764687     Source Type: Journal    
DOI: 10.1038/nature13420     Document Type: Article

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