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.
2.
NathwaniAC, RosalesC, McIntoshJ, et al.Long-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, 2011; 19:876–885.
3.
GeorgeLA, SullivanSK, GiermaszA, et al.Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med, 2017; 377:2215–2227.
4.
RangarajanS, WalshL, LesterW, et al.AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med, 2017; 377:2519–2530.
5.
MingozziF, HighKA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood, 2013; 122:23–36.
6.
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.
7.
MiesbachW, MeijerK, CoppensM, et al.Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood, 2018; 131:1022–1031.
8.
DayJW, MendellJR, MercuriE, et al.Clinical trial and postmarketing safety of onasemnogene abeparvovec therapy. Drug Saf, 2021; 44:1109–1119.
9.
ChandD, MohrF, McMillanH, et al.Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy. J Hepatol, 2021; 74:560–566.
10.
FeldmanAG, ParsonsJA, DutmerCM, et al. Subacute liver failure following gene replacement therapy for spinal muscular atrophy type 1. J Pediatr 2020;225:252.e1–258.e1.
Samelson-JonesBJ, FinnJD, FavaroP, et al.Timing of intensive immunosuppression impacts risk of transgene antibodies after AAV gene therapy in nonhuman primates. Mol Ther Methods Clin Dev, 2020; 17:1129–1138.
13.
ArrudaVR, Samelson-JonesBJ. Gene therapy for immune tolerance induction in hemophilia with inhibitors. J Thromb Haemost, 2016; 14:1121–1134.
14.
CrudeleJM, FinnJD, SinerJI, et al.AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood, 2015; 125:1553–1561.
15.
MingozziF, MeulenbergJJ, HuiDJ, et al.AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood, 2009; 114:2077–2086.
16.
RamageLE, AkyolM, FletcherAM, et al.Glucocorticoids acutely increase brown adipose tissue activity in humans, revealing species-specific differences in UCP-1 regulation. Cell Metab, 2016; 24:130–141.
17.
ShinJH, PanX, HakimCH, et al.Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy. Mol Ther, 2013; 21:750–757.
18.
ShinJH, YueY, SrivastavaA, et al.A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs. Hum Gene Ther, 2012; 23:202–209.
19.
YueY, PanX, HakimCH, et al.Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus. Hum Mol Genet, 2015; 24:5880–5890.
20.
Le GuinerC, MontusM, ServaisL, et al.Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients. Mol Ther, 2014; 22:1923–1935.
21.
Le GuinerC, ServaisL, MontusM, et al.Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nat Commun, 2017; 8:16105.
22.
VulinA, BarthelemyI, GoyenvalleA, et al.Muscle function recovery in golden retriever muscular dystrophy after AAV1–U7 exon skipping. Mol Ther, 2012; 20:2120–2133.
23.
WangL, WarzechaCC, KistnerA, et al.Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression. Mol Ther Methods Clin Dev, 2022; 24:292–305.
