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

Amit C. Nathwani*, John T. Gray, Catherine Y C Ng, Junfang Zhou, Yunyu Spence, Simon N. Waddington, Edward G D Tuddenham, Geoffrey Kemball-Cook, Jenny McIntosh, Mariette Boon-Spijker, Koen Mertens, Andrew M. Davidoff

*Corresponding author for this work

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Biochemistry, Genetics and Molecular Biology

Medicine and Dentistry