Globin gene transfer for the treatment of severe hemoglobinopathies: A paradigm for cell-based gene therapy

Journal of Gene Medicine

Volumn 4, Issue 2, 2002, Pages 113-121

  Sadelain, Michel ^a  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

Author keywords

thalassemia; Gene regulation; Hematopoietic stem cell; Lentiviral vector; Locus control region; Sickle cell disease

Indexed keywords

BETA GLOBIN; DEOXYRIBONUCLEASE I; GAG PROTEIN; HEMOGLOBIN A; HEMOGLOBIN ALPHA CHAIN; HEMOGLOBIN BETA CHAIN; HEMOGLOBIN F; HYDROXYUREA; LENTIVIRUS VECTOR; POL PROTEIN; RETROVIRUS VECTOR; VIRUS ENVELOPE PROTEIN; GLOBIN;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; BETA THALASSEMIA; CELL BASED GENE THERAPY; DISEASE SEVERITY; ERYTHROLEUKEMIA CELL; GENE EXPRESSION; GENE TRANSFER; GENETIC CODE; HEMOGLOBINOPATHY; HUMAN; NONHUMAN; NONVIRAL GENE THERAPY; OXYGEN TRANSPORT; PRIORITY JOURNAL; REVIEW; SICKLE CELL ANEMIA; SIDE EFFECT; STEM CELL; TRANSGENE; TRANSGENIC MOUSE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS GENOME; ANIMAL; BIOLOGICAL MODEL; CYTOLOGY; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; LENTIVIRINAE; METHODOLOGY; MOUSE;

ANIMALS; BETA-THALASSEMIA; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLOBINS; HEMOGLOBINOPATHIES; LENTIVIRUS; MICE; MICE, TRANSGENIC; MODELS, GENETIC; STEM CELLS; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 0036516375     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/jgm.266     Document Type: Review

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