Gene therapy for hemoglobinopathies: Progress and challenges

Translational Research

Volumn 161, Issue 4, 2013, Pages 293-306

  Dong, Alisa ^a   Rivella, Stefano ^a   Breda, Laura ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BT87Q VIRUS FUSION PROTEIN; BUSULFAN; CD34 ANTIGEN; CYCLOPHOSPHAMIDE; GRANULOCYTE COLONY STIMULATING FACTOR; HEMOGLOBIN; HEMOGLOBIN F; HEMOGLOBIN GAMMA CHAIN; HIGH MOBILITY GROUP A2 PROTEIN; LENTIVIRUS VECTOR; OLIGONUCLEOTIDE; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG; VIRUS FUSION PROTEIN;

3' UNTRANSLATED REGION; ALLOGENIC BONE MARROW TRANSPLANTATION; BETA THALASSEMIA; BINDING SITE; ENHANCER REGION; EXPRESSION VECTOR; GENE CONTROL; GENE TRANSFER; GENETIC HETEROGENEITY; GLOBIN GENE; GLOBIN SYNTHESIS; GRAFT VERSUS HOST REACTION; HEMOGLOBIN BLOOD LEVEL; HEMOGLOBINOPATHY; HUMAN; IRON CHELATION; NONHUMAN; PERIPHERAL BLOOD MONONUCLEAR CELL; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; REGULATOR GENE; REVIEW; RNA TRANSCRIPTION; RNA TRANSLATION; SICKLE CELL; SICKLE CELL ANEMIA; SINGLE NUCLEOTIDE POLYMORPHISM; STABLE EXPRESSION; STOICHIOMETRY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84875155964     PISSN: 19315244     EISSN: 18781810     Source Type: Journal    
DOI: 10.1016/j.trsl.2012.12.011     Document Type: Review

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