Genetic targeting of the albumin locus to treat hemophilia

New England Journal of Medicine

Volumn 374, Issue 13, 2016, Pages 1288-1290

  Davidoff, Andrew M ^a   Nathwani, Amit C ^b  

^a UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ALBUMIN; BLOOD CLOTTING FACTOR 9; PARVOVIRUS VECTOR;

ALB GENE; ALBUMIN BLOOD LEVEL; ARTICLE; CARCINOGENESIS; GENE EXPRESSION; GENE LOCUS; GENE PRODUCT; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HEMOPHILIA A; HEMOPHILIA B; HUMAN; HYPERTRANSAMINASEMIA; IMMUNOGENICITY; LIVER CELL; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; TROPISM; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE VECTOR; GENETICS; LIVER; MOUSE;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FACTOR IX; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMOPHILIA B; HUMANS; LIVER; MICE;

EID: 84962440587     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMcibr1600347     Document Type: Article

References (5)

1. Nathwani AC, Reiss UM, Tuddenham EGD, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994-2004.
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4. Barzel A, Paulk NK, Shi Y, et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature 2015;517:360-4.
5. Sharma R, Anguela XM, Doyon Y, et al. In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood 2015;126:1777-84.