CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

Science Translational Medicine

Volumn 9, Issue 372, 2017, Pages

  De Ravin, Suk See ^a   Li, Linhong ^b   Wu, Xiaolin ^c   Choi, Uimook ^a   Allen, Cornell ^b   Koontz, Sherry ^a   Lee, Janet ^a   Theobald Whiting, Narda ^a   Chu, Jessica ^a   Garofalo, Mary ^a   Sweeney, Colin ^a   Kardava, Lela ^a   Moir, Susan ^a   Viley, Angelia ^b   Natarajan, Pachai ^b   Su, Ling ^c   Kuhns, Douglas ^a   Zarember, Kol A ^a   Peshwa, Madhusudan V ^b   Malech, Harry L ^a  

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

^b MAXCYTE INC   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; CD45 ANTIGEN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE 2; SUPEROXIDE; CYBB PROTEIN, HUMAN; OLIGONUCLEOTIDE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE MARROW CELL; CELL DIFFERENTIATION; CELL MATURATION; CELL PROLIFERATION; CELL VIABILITY; CHRONIC GRANULOMATOUS DISEASE; CONTROLLED STUDY; CRISPR CAS SYSTEM; CYBB GENE; ENGRAFTMENT; ENZYME ACTIVITY; GENE; GENE EDITING; GENE FREQUENCY; GENE MUTATION; GENE SEQUENCE; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUMAN CELL; LYMPHOID CELL; MOUSE; NONHUMAN; PROTEIN EXPRESSION; PROTEIN FUNCTION; TARGETED GENE REPAIR; WHOLE EXOME SEQUENCING; X CHROMOSOME LINKED DISORDER; X LINKED CHRONIC GRANULOMATOUS DISEASE; ANIMAL; CYTOLOGY; DNA REPAIR; FEMALE; GENE THERAPY; GENETICS; METABOLISM; MUTAGENESIS; MUTATION; NONOBESE DIABETIC MOUSE; SCID MOUSE;

ANIMALS; ANTIGENS, CD34; CELL DIFFERENTIATION; CRISPR-CAS SYSTEMS; DNA REPAIR; FEMALE; GENETIC THERAPY; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELLS; HUMANS; MICE; MICE, INBRED NOD; MICE, SCID; MUTAGENESIS; MUTATION; NADPH OXIDASE 2; OLIGONUCLEOTIDES;

EID: 85010635762     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aah3480     Document Type: Article

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