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Comment
. 2011 May;19(5):826-7.
doi: 10.1038/mt.2011.73.

A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates

Affiliations
Comment

A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates

Randy J Chandler et al. Mol Ther. 2011 May.
No abstract available

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References

    1. Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D.et al. (2011Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins Mol Ther 19876–885. - PMC - PubMed
    1. Ponder KP. Hemophilia gene therapy: a holy grail found. Mol Ther. 2011;19:427–428. - PMC - PubMed
    1. Murphy SL., and, High KA. Gene therapy for haemophilia. Br J Haematol. 2008;140:479–487. - PMC - PubMed
    1. Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A.et al. (2000Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector Nat Genet 24257–261. - PubMed
    1. Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR.et al. (2003AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B Blood 1012963–2972. - PubMed