Gene therapy for monogenic disorders of the bone marrow

British Journal of Haematology

Volumn 171, Issue 2, 2015, Pages 155-170

  Ghosh, Sujal ^a,b   Thrasher, Adrian J ^a   Gaspar, H Bobby ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b HEINRICH HEINE UNIVERSITY   (Germany)

Author keywords

Gene therapy; Haemoglobinopathies; Metabolic disorders; Primary immunodeficiency

Indexed keywords

GAMMARETROVIRAL VECTOR; LENTIVIRUS VECTOR; PEGADEMASE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE; RETROVIRUS VECTOR; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; VIRUS VECTOR; ZINC FINGER NUCLEASE;

ADENOSINE DEAMINASE DEFICIENCY; BETA THALASSEMIA; BONE MARROW DISEASE; CHRONIC GRANULOMATOUS DISEASE; CRISPR CAS SYSTEM; DNA END JOINING REPAIR; ENZYME REPLACEMENT; EX VIVO GENE TRANSFER; FANCONI ANEMIA; GENE EXPRESSION REGULATION; GENE THERAPY; GENOTOXICITY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HUMAN; IMMUNE DEFICIENCY; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MONOGENIC DISORDER; NONHUMAN; PRIORITY JOURNAL; REVIEW; SICKLE CELL ANEMIA; WISKOTT ALDRICH SYNDROME; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

EID: 84942984045     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.13520     Document Type: Review

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