BrantlyM.L., ChulayJ.D., WangL., et al. (2009). Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc. Natl. Acad. Sci. USA, 106, 16363–16368.
2.
FerreiraV., TwiskJ., KwikkersK., et al. (2014). Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPLS447X) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy. Hum. Gene Ther., 25, 180–188.
3.
FlotteT.R., TrapnellB.C., HumphriesM., et al. (2011). Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum. Gene Ther., 22, 1239–1247.
4.
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.
5.
MuellerC., ChulayJ.D., TrapnellB.C., et al. (2013). Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J. Clin. Invest., 123, 5310–5318.
6.
NathwaniA.C., TuddenhamE.G., RangarajanS., et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med., 365, 2357–2365.
