Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

Science Translational Medicine

Volumn 8, Issue 360, 2016, Pages

  DeWitt, Mark A ^a,b   Magis, Wendy ^c   Bray, Nicolas L ^a,b   Wang, Tianjiao ^a,b   Berman, Jennifer R ^d   Urbinati, Fabrizia ^e   Heo, Seok Jin ^c   Mitros, Therese ^b   Muñoz, Denise P ^c   Boffelli, Dario ^c   Kohn, Donald B ^e   Walters, Mark C ^c,f   Carroll, Dana ^a,g   Martin, David I K ^c   Corn, Jacob E ^a,b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b University of California Berkeley   (United States)

^c CHILDREN S HOSPITAL OAKLAND RESEARCH INSTITUTE   (United States)

^d BIO RAD LABORATORIES   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f CHILDREN S HOSPITAL OAKLAND   (United States)

^g UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CAS9 PROTEIN; CD34 ANTIGEN; CELLULAR APOPTOSIS SUSCEPTIBILITY PROTEIN; GUIDE RNA; HEMOGLOBIN S; OLIGONUCLEOTIDE; SINGLE STRANDED DNA; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ARTICLE; CELL DIFFERENTIATION; CONTROLLED STUDY; ENGRAFTMENT; ERYTHROBLAST; EX VIVO STUDY; GENE MUTATION; GENE THERAPY; GENOME EDITING; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; K562 CELL LINE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SICKLE CELL ANEMIA; ADULT; ADULT STEM CELL; ANIMAL; CELL LINE; CRISPR CAS SYSTEM; GENE EDITING; GENETICS; KNOCKOUT MOUSE; METABOLISM; MUTATION; NONOBESE DIABETIC MOUSE; PROCEDURES; SCID MOUSE; SINGLE NUCLEOTIDE POLYMORPHISM; TRANSLATIONAL RESEARCH; XENOGRAFT;

ADULT; ADULT STEM CELLS; ANEMIA, SICKLE CELL; ANIMALS; CELL LINE; CRISPR-CAS SYSTEMS; GENE EDITING; HEMATOPOIETIC STEM CELLS; HEMOGLOBIN, SICKLE; HETEROGRAFTS; HUMANS; MICE; MICE, INBRED NOD; MICE, KNOCKOUT; MICE, SCID; MUTATION; POLYMORPHISM, SINGLE NUCLEOTIDE; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84991780675     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aaf9336     Document Type: Article

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