Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

Journal of Clinical Investigation

Volumn 126, Issue 10, 2016, Pages 3868-3878

  Brendel, Christian ^a,b   Guda, Swaroopa ^a,b   Renella, Raffaele ^a,b,c,h   Bauer, Daniel E ^a,b,c   Canver, Matthew C ^b   Kim, Young Jo ^a,b   Heeney, Matthew M ^a,b,c   Klatt, Denise ^d   Fogel, Jonathan ^a   Milsom, Michael D ^e   Orkin, Stuart H ^a,b,c,f,g   Gregory, Richard I ^a,b,g   Williams, David A ^a,b,c,g  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c DANA FARBER CANCER INSTITUTE   (United States)

^d HANNOVER MEDICAL SCHOOL   (Germany)

^e GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^f HOWARD HUGHES MEDICAL INSTITUTE   (United States)

^g HARVARD STEM CELL INSTITUTE   (United States)

^h LAUSANNE UNIVERSITY HOSPITAL   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

BCL11A PROTEIN; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN; MICRORNA; REGULATOR PROTEIN; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG; BCL11A PROTEIN, HUMAN; CARRIER PROTEIN; CD34 ANTIGEN; NUCLEAR PROTEIN; SMALL INTERFERING RNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BCL11A GENE; CELL CYCLE G2 PHASE; CELL CYCLE S PHASE; CONTROLLED STUDY; CYTOTOXICITY; DISEASE MARKER; ERYTHROCYTE; ERYTHROID CELL; GENE; GENE SILENCING; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOLYTIC ANEMIA; HUMAN; HUMAN CELL; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; RETICULOCYTOSIS; SICKLE CELL; SICKLE CELL ANEMIA; ANEMIA, SICKLE CELL; ANIMAL; C57BL MOUSE; CELL CULTURE; CELL LINEAGE; GENE THERAPY; GENETICS; GRAFT VS HOST DISEASE; HEMATOPOIESIS; METABOLISM; NONOBESE DIABETIC MOUSE; PHYSIOLOGY; SCID MOUSE;

ANEMIA, SICKLE CELL; ANIMALS; ANTIGENS, CD34; CARRIER PROTEINS; CELL LINEAGE; CELLS, CULTURED; GENE KNOCKDOWN TECHNIQUES; GENETIC THERAPY; GRAFT VS HOST DISEASE; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; MICE, INBRED C57BL; MICE, INBRED NOD; MICE, SCID; NUCLEAR PROTEINS; PHENOTYPE; RNA, SMALL INTERFERING;

EID: 84991665780     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI87885     Document Type: Article

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