Developments in the treatment of hemophilia B: Focus on emerging gene therapy

Application of Clinical Genetics

Volumn 6, Issue , 2013, Pages 91-101

  Cancio, Maria I ^a   Reiss, Ulrike M ^a   Nathwani, Amit C ^b   Davidoff, Andrew M ^a   Gray, John T ^a  

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

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Adeno associated virus; Adenovirus gene therapy; Factor IX deficiency; Hemophilia B

Indexed keywords

GENE THERAPY AGENT; PARVOVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9;

ADAPTIVE IMMUNITY; AMINO ACID SEQUENCE; ANTIGEN PRESENTING CELL; CATALYSIS; CD8+ T LYMPHOCYTE; CYTOTOXIC T LYMPHOCYTE; DISSEMINATED INTRAVASCULAR CLOTTING; DRUG COST; DRUG MEGADOSE; DRUG POTENCY; DRUG SAFETY; DRUG TOLERABILITY; EPISOME; GENE EXPRESSION; GENE THERAPY; HEMOPHILIA B; HUMAN; HYPERTRANSAMINASEMIA; INNATE IMMUNITY; LIVER TOXICITY; MAJOR HISTOCOMPATIBILITY COMPLEX; MUSCLE BIOPSY; PATHOPHYSIOLOGY; PROTEIN EXPRESSION; REVIEW; TRANSGENE; VIRAL GENE THERAPY;

EID: 84886879931     PISSN: None     EISSN: 1178704X     Source Type: Journal    
DOI: 10.2147/TACG.S31928     Document Type: Review

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