Current status of globin gene therapy for the treatment of β-thalassaemia

British Journal of Haematology

Volumn 141, Issue 3, 2008, Pages 335-345

  Lisowski, Leszek ^b,c   Sadelain, Michel ^a,b  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^b Department of Obstetrics Gynecology   (United States)

^c Weill Cornell Graduate School of Biomedical Sciences   (United States)

Author keywords

Erythroid specific expression; Globin gene expression; Lentiviral vectors; Stem cell therapy; Thalassaemia

Indexed keywords

BETA GLOBIN; LENTIVIRUS VECTOR; RETROVIRUS VECTOR;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; BETA THALASSEMIA; CELL DIFFERENTIATION; CONFERENCE PAPER; DRUG DESIGN; DRUG EFFICACY; DRUG SAFETY; GENE DELETION; GENE EXPRESSION; GENE SILENCING; GENETIC REGULATION; HEMOGLOBINOPATHY; HLA MATCHING; HUMAN; IN VIVO STUDY; INSULATOR ELEMENT; INTRON; IRON OVERLOAD; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; STEM CELL GENE THERAPY; TRANSCRIPTION REGULATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

BETA-THALASSEMIA; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLOBINS; HUMANS; INSULATOR ELEMENTS; LENTIVIRUS; RETROVIRIDAE;

EID: 42149153870     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2008.07098.x     Document Type: Conference Paper

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