CouzinJ, KaiserJ. Gene therapy. As Gelsinger case ends, gene therapy suffers another blow. Science, 2005; 307:1028.
8.
MingozziF, HighKA. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet, 2011; 12:341–355.
9.
AronsonSJ, VeronP, CollaudF, et al.Prevalence and relevance of pre-existing anti-adeno-associated virus immunity in the context of gene therapy for Crigler-Najjar syndrome. Hum Gene Ther, 2019; 30:1297–1305.
10.
JauzeL, MonteilletL, MithieuxG, et al.Challenges of gene therapy for the treatment of glycogen storage diseases type I and type III. Hum Gene Ther, 2019; 30:1263–1273.
11.
ColellaP, MingozziF. Gene therapy for Pompe disease: the time is now. Hum Gene Ther, 2019; 30:1245–1262.
12.
Grisch-ChanHM, SchwankG, HardingCO, ThonyB. State-of-the-art 2019 on gene therapy for phenylketonuria. Hum Gene Ther, 2019; 30:1274–1283.
13.
ChandlerRJ, VendittiCP. Gene Therapy for methylmalonic acidemia: Past, present, and future. Hum Gene Ther, 2019; 30:1236–1244.
14.
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.
15.
NaldiniL. Gene therapy returns to centre stage. Nature, 2015; 526:351–360.
16.
Sii-FeliceK, Castillo PadillaJ, RelouzatF, et al.Enhanced transduction of Macaca fascicularis hematopoietic cells with chimeric lentiviral vectors. Hum Gene Ther, 2019; 30:1306–1323.
17.
MilaniM, AnnoniA, MoalliF, et al.Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates. Sci Transl Med, 2019; 11:eaav7325.
ColellaP, RonzittiG, MingozziF. Emerging issues in AAV-mediated in vivo gene therapy. Mol Ther Methods Clin Dev, 2018; 8:87–104.
20.
CallejasD, MannCJ, AyusoE, et al.Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy. Diabetes, 2013; 62:1718–1729.
