Hemophilia B Gene Therapy in Humans Shows Promise | Zendy

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Hemophilia B Gene Therapy in Humans Shows Promise

Author(s) -

Andrew D. Johnson

Publication year - 2012

Publication title -

circulation cardiovascular genetics

Language(s) - English

Resource type - Journals

eISSN - 1942-325X

pISSN - 1942-3268

DOI - 10.1161/circgenetics.112.963231

Subject(s) - genetic enhancement , medicine , factor ix , adverse effect , immune system , heart failure , gene delivery , vector (molecular biology) , framingham heart study , disease , immunology , intensive care medicine , bioinformatics , framingham risk score , gene , biology , genetics , recombinant dna

1. Nathwani AC, Gray JT, Ng CYC, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Gene Therapy. 2006;107:2653–2661. 2. Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM. Long-term safety and efficacy following systemic administration of a selfcomplementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Therapy. 2011;19:876–885.

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