Targeted gene addition in human CD34 + hematopoietic cells for correction of X-linked chronic granulomatous disease

Nature Biotechnology

Volumn 34, Issue 4, 2016, Pages 424-429

  De Ravin, Suk See ^a   Reik, Andreas ^b   Liu, Pei Qi ^b   Li, Linhong ^c   Wu, Xiaolin ^d   Su, Ling ^d   Raley, Castle ^d   Theobald, Narda ^a   Choi, Uimook ^a   Song, Alexander H ^b   Chan, Andy ^b   Pearl, Jocelynn R ^b   Paschon, David E ^b   Lee, Janet ^a   Newcombe, Hannah ^a   Koontz, Sherry ^a   Sweeney, Colin ^a   Shivak, David A ^b   Zarember, Kol A ^a   Peshwa, Madhusudan V ^d   Gregory, Philip D ^b,e   Urnov, Fyodor D ^b   Malech, Harry L ^a   more..

^a NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^b SANGAMO BIOSCIENCES INC   (United States)

^c MAXCYTE INC   (United States)

^d FREDERICK NATIONAL LABORATORY FOR CANCER RESEARCH   (United States)

^e Blubird Bio   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELLS; CYTOLOGY; GENE THERAPY; GENES; MAMMALS; VIRUSES;

ADENO-ASSOCIATED VIRUS; ELECTROPORATION; ENGRAFTMENT; GENETICALLY MODIFIED; HEMATOPOIETIC CELL; NADPH OXIDASE; PROGENITOR CELL; ZINC FINGER NUCLEASE;

STEM CELLS;

MESSENGER RNA; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; ZINC FINGER NUCLEASE; CD34 ANTIGEN;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ARTICLE; CD34 SELECTION; CHRONIC GRANULOMATOUS DISEASE; CONTROLLED STUDY; ELECTROPORATION; ENGRAFTMENT; ENZYME ACTIVITY; EX VIVO STUDY; GENE LOCUS; GENE MUTATION; GENE TARGETING; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; MOUSE; NEUTROPHIL; NONHUMAN; PRIORITY JOURNAL; TRANSGENE; X CHROMOSOME LINKED DISORDER; ANIMAL; CELL CULTURE; CHEMISTRY; CYTOLOGY; GENE THERAPY; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; PROCEDURES; TRANSGENIC MOUSE;

ANIMALS; ANTIGENS, CD34; CELLS, CULTURED; GENETIC THERAPY; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; MICE; MICE, TRANSGENIC;

EID: 84963516618     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.3513     Document Type: Article

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