Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

doi:10.1182/blood-2012-10-462200

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Article

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

Blood - 25 Apr 2013

McIntosh Jenny, Lenting Peter J, Rosales Cecilia, Lee Doyoung, Rabbanian Samira, Raj Deepak, Patel Nishil, Tuddenham Edward G D, Christophe Olivier D, McVey John H, Waddington Simon, Nienhuis Arthur W, Gray John T, Fagone Paolo, Mingozzi Federico, Zhou Shang-Zhen, High Katherine A, Cancio Maria, Ng Catherine Y C, Zhou Junfang, Morton Christopher L, Davidoff Andrew M, Nathwani Amit C

Abstract excerpt

Recombinant adeno-associated virus (rAAV) vectors encoding human factor VIII (hFVIII) were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV-expression cassette (rAAV-HLP-codop-hFVIII-N6) containing a codon-optimized hFVIII cDNA in which a 226 amino acid (aa) B-domain spacer replaced the entire B domain and a hybrid liver-specific promoter (HLP) mediated 10-fold higher hFVIII levels in...

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PMID: 23426947
DOI: 10.1182/blood-2012-10-462200
PMCID: PMC3637010

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