The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells

Human Gene Therapy

Volumn 23, Issue 1, 2012, Pages 15-31

  Papanikolaou, Eleni ^a,b   Georgomanoli, Maria ^a,b   Stamateris, Evangelos ^a,b   Panetsos, Fottes ^c   Karagiorga, Markisia ^d   Tsaftaridis, Panagiotis ^e   Graphakos, Stelios ^b   Anagnou, Nicholas P ^a,b  

^a BIOMEDICAL RESEARCH FOUNDATION   (Greece)

^b UNIVERSITY OF ATHENS MEDICAL SCHOOL   (Greece)

^c Deparment of Biodiagnostics   (Greece)

^d Haemophilia Haemostasis Unit   (Greece)

^e LAIKO GENERAL HOSPITAL   (Greece)

Author keywords

[No Author keywords available]

Indexed keywords

117 HEREDITARY PERSISTENCE OF FETAL HEMOGLOBIN; CD34 ANTIGEN; GAMMA GLOBIN; GGHI SELF INACTIVATING LENTIVIRUS VECTOR; HEMOGLOBIN F; HEREDITARY PERSISTENCE OF FETAL HEMOGLOBIN 2 ENHANCER; LENTIVIRUS VECTOR; UNCLASSIFIED DRUG;

APOPTOSIS; ARTICLE; BETA THALASSEMIA; CELL POPULATION; COPY NUMBER VARIATION; ERYTHROBLAST; ERYTHROID CELL; ERYTHROPOIESIS; FEASIBILITY STUDY; GENE THERAPY; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; POINT MUTATION; REGULATOR GENE; SIGNAL TRANSDUCTION;

ANTIGENS, CD34; APOPTOSIS; BETA-THALASSEMIA; CHROMATOGRAPHY, HIGH PRESSURE LIQUID; CLONING, MOLECULAR; ENHANCER ELEMENTS, GENETIC; ERYTHROID CELLS; ERYTHROPOIESIS; FETAL HEMOGLOBIN; GAMMA-GLOBINS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEK293 CELLS; HEMATOPOIETIC STEM CELLS; HUMANS; LENTIVIRUS; MEMBRANE GLYCOPROTEINS; PLASMIDS; POINT MUTATION; PROMOTER REGIONS, GENETIC; TRANSFECTION; VESICULAR STOMATITIS INDIANA VIRUS; VIRAL ENVELOPE PROTEINS; VIRUS INACTIVATION;

EID: 84855909237     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.048     Document Type: Article

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