A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction

Annals of Hematology

Volumn 91, Issue 8, 2012, Pages 1201-1213

  Zuccato, Cristina ^a   Breda, Laura ^b   Salvatori, Francesca ^a   Breveglieri, Giulia ^a   Gardenghi, Sara ^b   Bianchi, Nicoletta ^a   Brognara, Eleonora ^a   Lampronti, Ilaria ^a   Borgatti, Monica ^a   Rivella, Stefano ^b   Gambari, Roberto ^a  

^a UNIVERSITY OF FERRARA   (Italy)

^b WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

thalassemia; Erythroid progenitor cells; Gene therapy; HbF induction; Lentiviral vectors

Indexed keywords

ALPHA GLOBIN; BETA GLOBIN; GAMMA GLOBIN; HEMOGLOBIN A; HEMOGLOBIN F; LENTIVIRUS VECTOR; MESSENGER RNA; MITHRAMYCIN;

ADULT; ARTICLE; BETA GLOBIN GENE; BETA THALASSEMIA; CELL CLONE; CELL STRAIN K 562; CONTROLLED STUDY; ERYTHROID PRECURSOR CELL; FETUS; GENE; GENE EXPRESSION; HEMOGLOBIN SYNTHESIS; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HUMAN; HUMAN CELL; IN VITRO GENE TRANSFER; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN INDUCTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADULT; ANTIBIOTICS, ANTINEOPLASTIC; BETA-GLOBINS; BETA-THALASSEMIA; CELLS, CULTURED; COMBINED MODALITY THERAPY; ERYTHROID PRECURSOR CELLS; FETAL HEMOGLOBIN; GENE THERAPY; GENE TRANSFER TECHNIQUES; HEK293 CELLS; HEMOGLOBIN A; HUMANS; K562 CELLS; PLICAMYCIN;

EID: 84864982332     PISSN: 09395555     EISSN: 14320584     Source Type: Journal    
DOI: 10.1007/s00277-012-1430-5     Document Type: Article

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