Haemophilia gene therapy: Progress and challenges

Blood Reviews

Volumn 29, Issue 5, 2015, Pages 321-328

  Lheriteau, Elsa ^a,b   Davidoff, Andrew M ^d   Nathwani, Amit C ^a,b,c  

^a ROYAL FREE LONDON NHS FOUNDATION TRUST   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c NHS BLOOD AND TRANSPLANT   (United Kingdom)

^d ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

Factor IX; Factor VIII; Gene therapy; Haemophilia; Vectors

Indexed keywords

ADENOVIRUS VECTOR; LENTIVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 9;

ADENO ASSOCIATED VIRUS; ARTICLE; CODON USAGE; CYTOTOXIC T LYMPHOCYTE; DISEASE SEVERITY; DNA METHYLATION; DRUG EFFICACY; DRUG MEGADOSE; DRUG RESPONSE; DRUG SAFETY; FIX GENE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HEALTH CARE COST; HEMOPHILIA A; HEMOPHILIA B; HUMAN; HUMORAL IMMUNITY; IMMUNE RESPONSE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; PROTEIN GLYCOSYLATION; PROTEIN SECRETION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS GENE; PROCEDURES;

GENETIC THERAPY; HEMOPHILIA A; HUMANS;

EID: 84941260214     PISSN: 0268960X     EISSN: 15321681     Source Type: Journal    
DOI: 10.1016/j.blre.2015.03.002     Document Type: Article

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