FlotteTR, TrapnellBC, HumphriesM, et al.Phase 2 clinical trial of a recombinant adenoassociated viral vector expressing α1-antitrypsin: interim results. Hum Gene Ther, 2011; 22:1239–1247.
2.
GaudetD, MéthotJ, DéryS, et al.Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther, 2013; 20:361–369.
3.
NathwaniAC, ReissUM, TuddenhamEG, et al.Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med, 2014; 371:1994–2004.
4.
MannoCS, PierceGF, ArrudaVR, et al.Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med, 2006; 12:342–347.
5.
MingozziF, MausMV, HuiDJ, et al.CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med, 2007; 13:419–422.
6.
TarantalAF, LeeCC, MartinezML, et al.Systemic and persistent muscle gene expression in rhesus monkeys with a liver de-targeted adeno-associated virus vector. Hum Gene Ther, 2017; 28:385–391.
7.
AsokanA, ConwayJC, PhillipsJL, et al.Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol, 2010; 28:79–82.
8.
PacakCA, MahCS, ThattaliyathBD, et al.Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res, 2006; 99:e3–9.
9.
InagakiK, FuessS, StormTA, et al.Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther, 2006; 14:45–53.
10.
VandenDriesscheT, ThorrezL, Acosta-SanchezA, et al.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, 2007; 5:16–24.