MiRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

Molecular Therapy

Volumn 23, Issue 9, 2015, Pages 1465-1474

  Guda, Swaroopa ^a,b   Brendel, Christian ^a,b   Renella, Raffaele ^a,b,c   Du, Peng ^a   Bauer, Daniel E ^a,b,c   Canver, Matthew C ^b   Grenier, Jennifer K ^d   Grimson, Andrew W ^e   Kamran, Sophia C ^b,f   Thornton, James ^a   De Boer, Helen ^a   Root, David E ^d   Milsom, Michael D ^g   Orkin, Stuart H ^a,b,c,f   Gregory, Richard I ^a   Williams, David A ^a,b,c  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c DANA FARBER CANCER INSTITUTE   (United States)

^d BROAD INSTITUTE OF MIT AND HARVARD   (United States)

^e Department of Molecular Biology and Genetics   (United States)

^f HOWARD HUGHES MEDICAL INSTITUTE   (United States)

^g GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

BCL11A PROTEIN; DNA DIRECTED RNA POLYMERASE III; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN; LENTIVIRUS VECTOR; MICRORNA; REPRESSOR PROTEIN; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG; BCL11A PROTEIN, HUMAN; CARRIER PROTEIN; NUCLEAR PROTEIN; SMALL INTERFERING RNA;

ANIMAL CELL; ARTICLE; CELL LINEAGE; CONTROLLED STUDY; CYTOTOXICITY; EMBEDDING; GENE EXPRESSION; GENE SILENCING; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; INTERMETHOD COMPARISON; MAMMAL CELL; MOUSE; NONHUMAN; PROMOTER REGION; PROTEIN EXPRESSION; PROTEIN INDUCTION; RNA INTERFERENCE; RNA PROCESSING; RNA SEQUENCE; RNA STRUCTURE; ANIMAL; BIOSYNTHESIS; CELL LINE; CHEMISTRY; CONFORMATION; CYTOLOGY; ERYTHROID CELL; GENE ORDER; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; METABOLISM; NUCLEOTIDE SEQUENCE; RETROVIRIDAE;

ANIMALS; BASE SEQUENCE; CARRIER PROTEINS; CELL LINE; CELL LINEAGE; ERYTHROID CELLS; FETAL HEMOGLOBIN; GAMMA-GLOBINS; GENE EXPRESSION; GENE KNOCKDOWN TECHNIQUES; GENE ORDER; GENE SILENCING; GENETIC VECTORS; HUMANS; MICE; MICRORNAS; NUCLEAR PROTEINS; NUCLEIC ACID CONFORMATION; PROMOTER REGIONS, GENETIC; RETROVIRIDAE; RNA INTERFERENCE; RNA, SMALL INTERFERING; TRANSDUCTION, GENETIC;

EID: 84940724132     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.113     Document Type: Article

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