BainbridgeJWB, MehatMS, SundaramV, et al.Long-term effect of gene therapy on Leber's congenital amaurosis. N Engl J Med, 2015; 372:1887–1897.
2.
Hacein-Bey-AbinaS, HauerJ, LimA, et al.Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med, 2010; 363:355–364.
3.
NathwaniAC, TuddenhamEGD, RangarajanS, et al.Adenovirus-associated virus vector–mediated gene transfer in hemophilia B. N Engl J Med, 2011; 365:2357–2365.
4.
Ylä-HerttualaS. Endgame: Glybera finally recommended for approval as the first gene therapy drug in the European Union. Mol Ther, 2012; 20:1831–1832.
5.
AiutiA, RoncaroloMG, NaldiniL. Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products. EMBO Mol Med, 2017; 9:737–740.
