Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients

EMBO Molecular Medicine

Volumn 2, Issue 8, 2010, Pages 315-328

  Roselli, Emanuela Anna ^a   Mezzadra, Riccardo ^a   Frittoli, Marta Claudia ^a   Maruggi, Giulietta ^b   Biral, Erika ^c   Mavilio, Fulvio ^b,c   Mastropietro, Fabrizio ^d   Amato, Antonio ^d   Tonon, Giovanni ^c   Refaldi, Chiara ^e   Cappellini, Maria Domenica ^e   Andreani, Marco ^f   Lucarelli, Guido ^f   Roncarolo, Maria Grazia ^a,c,g   Marktel, Sarah ^c   Ferrari, Giuliana ^a,g  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b UNIVERSITY OF MODENA AND REGGIO EMILIA   (Italy)

^c SAN RAFFAELE HOSPITAL   (Italy)

^d Associazione Nazionale Microcitemie Italia ANMI ONLUS   (Italy)

^e UNIVERSITY OF MILAN   (Italy)

^f TOR VERGATA UNIVERSITY HOSPITAL   (Italy)

^g VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

Author keywords

Gene therapy; Haematopoietic stem cells; Haemoglobin; Lentiviral vectors; Locus control region

Indexed keywords

BETA GLOBIN; GLOBE LENTIVIRUS VECTOR; HEMOGLOBIN A; LENTIVIRUS VECTOR; UNCLASSIFIED DRUG;

ADOLESCENT; APOPTOSIS; ARTICLE; BONE MARROW TRANSPLANTATION; CD34 SELECTION; CHILD; CLINICAL ARTICLE; DRUG EFFICACY; DRUG SAFETY; ERYTHROPOIESIS; FEMALE; GENE EXPRESSION PROFILING; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HEMOGLOBIN SYNTHESIS; HUMAN; HUMAN CELL; MALE; PRESCHOOL CHILD; PRIORITY JOURNAL; SCHOOL CHILD; SIGNAL TRANSDUCTION; THALASSEMIA MAJOR; TRANSCRIPTION REGULATION; AUTOTRANSPLANTATION; BETA-THALASSEMIA; BIOSYNTHESIS; CELL CULTURE; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; LENTIVIRINAE; METABOLISM; PROCEDURES;

ADOLESCENT; BETA-THALASSEMIA; CELLS, CULTURED; CHILD; CHILD, PRESCHOOL; FEMALE; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HEMOGLOBIN A; HUMANS; LENTIVIRUS; MALE; TRANSDUCTION, GENETIC; TRANSPLANTATION, AUTOLOGOUS; GENETIC THERAPY;

EID: 77956949262     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.1002/emmm.201000083     Document Type: Article

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