Recent trends in the gene therapy of β-thalassemia

Journal of Blood Medicine

Volumn 6, Issue , 2015, Pages 69-85

  Finotti, Alessia ^a,b   Breda, Laura ^c   Lederer, Carsten W ^d,e   Bianchi, Nicoletta ^a,b   Zuccato, Cristina ^a,b   Kleanthous, Marina ^d,e   Rivella, Stefano ^c   Gambari, Roberto ^a,b  

^a UNIVERSITY OF FERRARA   (Italy)

^b Associazione Veneta per la Lotta Alla Talassemia   (Italy)

^c WEILL CORNELL MEDICAL COLLEGE   (United States)

^d CYPRUS INSTITUTE OF NEUROLOGY AND GENETICS   (Cyprus)

^e Cyprus School of Molecular Medicine   (Cyprus)

Author keywords

CRISPR; Gene therapy; Genome editing; HbF induction; Induced pluripotent stem cells; TALEN; Thalassemia; Transcription factors; ZFN

Indexed keywords

CASPASE 9; CD34 ANTIGEN; DNA DIRECTED RNA POLYMERASE III; ERYTHROID KRUPPEL LIKE FACTOR; HEMOGLOBIN BETA CHAIN; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN; HIGH MOBILITY GROUP A2 PROTEIN; HYDROXYUREA; KRUPPEL LIKE FACTOR 4; LENTIVIRUS VECTOR; MESSENGER RNA; MICRORNA 15A; MICRORNA 223; MITHRAMYCIN; OCTAMER TRANSCRIPTION FACTOR 1; OCTAMER TRANSCRIPTION FACTOR 4; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; ZINC FINGER NUCLEASE;

BETA THALASSEMIA; CLINICAL TRIAL (TOPIC); CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; ERYTHROID PRECURSOR CELL; FOLLOW UP; GENE ADDITION; GENE MUTATION; GENE OVEREXPRESSION; GENE THERAPY; GENOME EDITING; GENOTOXICITY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUMAN EMBRYONIC STEM CELL; IN VITRO STUDY; IN VIVO STUDY; NONHUMAN; PERSONALIZED MEDICINE; PLURIPOTENT STEM CELL; PROTEIN EXPRESSION; REVIEW; RNA INTERFERENCE; TRANSGENE;

EID: 85006186592     PISSN: None     EISSN: 11792736     Source Type: Journal    
DOI: 10.2147/JBM.S46256     Document Type: Review

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