AxelrodJ.H., ReadM.S., BrinkhousK.M., VermaI.M.1990. Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilic dogs. Proc. Natl. Acad. Sci. U.S.A., 87:5173–5177.
2.
GaoG.P., AlviraM.R., WangL.et al.2002. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A., 99:11854–11859.
3.
HighK.A.2011. Gene therapy for haemophilia: a long and winding road. J. Thromb. Haemost., 9,Suppl 1:2–11.
4.
JiangH., PierceG.F., OzeloM.C.et al.2006. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol. Ther., 14:452–455.
5.
KayM.A., HighK.1999. Gene therapy for the hemophilias. Proc. Natl. Acad. Sci. U.S.A., 96:9973–9975.
6.
KayM.A., MannoC.S., RagniM.V.et al.2000. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet., 24:257–261.
7.
MannoC.S., PierceG.F., ArrudaV.R.et al.2006. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med., 12:342–347.
8.
MátraiJ., ChuahM.K., VandenDriesscheT.2010. Preclinical and clinical progress in hemophilia gene therapy. Curr. Opin. Hematol., 17:387–392.
9.
McCartyD.M., MonahanP.E., SamulskiR.J.2001. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther., 8:1248–1254.
10.
McCartyD.M., FuH., MonahanP.E.et al.2003. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther., 10:2112–2118.
11.
MingozziF., LiuY.L., DobrzynskiE.et al.2003. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J. Clin. Invest., 111:1347–1356.
12.
MingozziF., MausM.V., HuiD.J.et al.2007. CD8+ T-cell responses to adeno-associated virus capsid in humans. Nat. Med., 13:419–422.
13.
NathwaniA.C., GrayJ.T., NgC.Y.et al.2006. 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. Blood, 107:2653–2661.
14.
NathwaniA., TuddenhamE.G.D., RangarajanS.et al.2011. Adenovirus-associated virus vector–mediated gene transfer in hemophilia B.N. Engl. J. Med.[Epub ahead of print]
15.
VandenbergheL.H., WangL., SomanathanS.et al.2006. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat. Med., 12:967–971.
16.
VandenDriesscheT., ThorrezL., Acosta-SanchezA.et al.2007. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy. J. Thromb. Haemost., 5:16–24.
17.
WardN.J., BuckleyS.M., WaddingtonS.N.et al.2011. Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood, 117:798–807.