JacobsonS.G., CideciyanA.V., RatnakaramR.et al.2012. Gene therapy for Leber congenital amaurosis caused by RPE65 mutations: Safety and efficacy in 15 children and adults followed up to 3 years. Arch. Ophthalmol., 130:9–24.
2.
KaiserJ.2011. Clinical research: Gene therapists celebrate a decade of progress. Science, 334:29–30.
3.
KohnD.B.2010. Update on gene therapy for immunodeficiencies. Clin. Immunol., 135:247–254.
4.
NathwaniA.C., TuddenhamE.G., RangarajanS.et al.2011. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med., 365:2357–2365.
5.
PonderK.P.2011. Merry Christmas for patients with hemophilia B. N. Eng. J. Med., 365:2424–2425.
6.
SilverJ.N., FlotteT.R.2008. Towards a rAAV-based gene therapy for ADA-SCID: From ADA deficiency to current and future treatment strategies. Pharmacogenomics, 9:947–968.
