WilliamsDA, LemischkaIR, NathanDG, et al.Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse. Nature, 1984; 310:476–480.
2.
NaldiniL, BlömerU, GallayP, et al.In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science, 1996; 272:263–267.
3.
MiyoshiH, SmithKA, MosierDE, et al.Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science, 1999; 283:682–686.
4.
TakahashiK, YamanakaS. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell, 2006; 126:663–676.
5.
GancbergD. Twenty years of European Union support to gene therapy and gene transfer. Hum Gene Ther, 2017; 28:951–953.
6.
BennettJ, WellmanJ, MarshallKA, et al.Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on Phase 1 trial. Lancet, 2016; 388:661–672.
7.
NathwaniAC, TuddenhamEG, RangarajanS, et al.Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med, 2011; 365:2357–2365.
8.
RangarajanS, WalshL, LesterW, et al.AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med, 2017; 377:2519–2530.
9.
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.
10.
CantoreA, NairN, Della ValleP, et al.Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Blood, 2012; 120:4517–4520.
11.
MendellJR, Al-ZaidyS, ShellR, et al.Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med, 2017; 377:1713–1722.
12.
RíoP, NavarroS, BuerenJ. Advances in the gene therapy of Fanconi anemia. Hum Gene Ther, 2018; 29:1114–1123.
13.
RibeilJA, Hacein-Bey-AbinaS, PayenE, et al.Gene therapy in a patient with sickle cell disease. N Engl J Med, 2017; 376:848–855.
14.
ThompsonAA, WaltersMC, KwiatkowskiJ, et al.Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med, 2018; 378:1479–1493.
BlaeseRM, CulverKW, MillerAD, et al.T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science, 1995; 270:475–480.
17.
JuneCH, O'ConnorRS, KawalekarOU, et al.CAR T cell immunotherapy for human cancer. Science, 2018; 359:1361–1365.
18.
CongL, RanFA, CoxD, et al.Multiplex genome engineering using CRISPR/Cas systems. Science, 2013; 339:819–823.
19.
MaliP, YangL, EsveltKM, et al.RNA-guided human genome engineering via Cas9. Science, 2013; 339:823–826.
20.
Hacein-Bey-AbinaS, GarrigueA, WangGP, et al.Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest, 2008; 118:3132–3142.
21.
WangJ, ExlineCM, DeClercqJJ, et al.Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. Nat Biotechnol, 2015; 33:1256–1263.
22.
KosickiM, TombergK, BradleyA. Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements. Nat Biotechnol, 2018; 36:765–771.
23.
AmoasiiL, HildyardJCW, LiH, et al.Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Science, 2018Aug30 [Epub ahead of print]; DOI: 10.1126/science.aau1549.