Cavazzana-CalvoM., Hacein-BeyS., de Saint BasileG., GrossF., YvonE., NusbaumP., SelzF., HueC., CertainS., CasanovaJ.L., BoussoP., DeistF.L., FischerA.2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science, 288:669–672.
5.
Hacein-Bey-AbinaS., GarrigueA., WangG.P., SoulierJ., LimA., MorillonE., ClappierE., CaccavelliL., DelabesseE., BeldjordK., AsnafiV., MacIntyreE., Dal CortivoL., RadfordI., BrousseN., SigauxF., MoshousD., HauerJ., BorkhardtA., BelohradskyB.H., WintergerstU., VelezM.C., LeivaL., SorensenR., WulffraatN., BlancheS., BushmanF.D., FischerA., Cavazzana-CalvoM.2008. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest., 18:3132–3142.
6.
HollisR.P., NightingaleS.J., WangX., PepperK.A., YuX.J., BarskyL., CrooksG.M., KohnD.B.2006. Stable gene transfer to human CD34+ hematopoietic cells using the Sleeping Beauty transposon. Exp. Hematol., 34:1333–1343.
7.
IvicsZ., HackettP.B., PlasterkR.H., IzsvakZ.1997. Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell, 91:501–510.
8.
KohnD.B., CandottiF.2009. Gene therapy fulfilling its promise. N. Engl. J. Med., 360:518–521.
9.
LombardoA., GenoveseP., BeausejourC.M., ColleoniS., LeeY.L., KimK.A., AndoD., UrnovF.D., GalliC., GregoryP.D., HolmesM.C., NaldiniL.2007. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat. Biotechnol., 25:1298–1306.
10.
MátésL., ChuahM.K., BelayE., JerchowB., ManojN., Acosta-SanchezA., GrzelaD.P., SchmittA., BeckerK., MatraiJ., MaL., Samara-KukoE., GysemansC., PryputniewiczD., MiskeyC., FletcherB., VandenDriesscheT., IvicsZ., IzsvákZ.2009. Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates. Nat. Genet., 41:753–761.
11.
ModlichU., BaumC.2009. Preventing and exploiting the oncogenic potential of integrating gene vectors. J. Clin. Invest., 119:755–758.
12.
MontiniE., CesanaD., SchmidtM., SanvitoF., BartholomaeC.C., RanzaniM., BenedicentiF., SergiL.S., AmbrosiA., PonzoniM., DoglioniC., Di SerioC., von KalleC., NaldiniL.2009. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J. Clin. Invest., 119:964–975.
13.
NaldiniL.2009. A comeback for gene therapy. Science, 326:805–806.
14.
SinghH., ManuriP.R., OlivaresS., DaraN., DawsonM.J., HulsH., HackettP.B., KohnD.B., ShpallE.J., ChamplinR.E., CooperL.J.2008. Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer Res., 68:2961–2971.
15.
SumiyoshiT., HoltN.G., HollisR.P., GeS., CannonP.M., CrooksG.M., KohnD.B.2009. Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells using the Sleeping Beauty transposon system. Hum. Gene Ther., 20:1607–1626
VandenDriesscheT., IvicsZ., IzsvákZ., ChuahM.K.2009. Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells. Blood, 114:1461–1468.
18.
WaliskoO., SchornA., RolfsF., DevarajA., MiskeyC., IzsvákZ., IvicsZ.2008. Transcriptional activities of the Sleeping Beauty transposon and shielding its genetic cargo with insulators. Mol. Ther., 16:359–369.
19.
XueX., HuangX., NodlandS.E., MátésL., MaL., IzsvákZ., IvicsZ., LeBienT.W., McIvorR.S., WagnerJ.E., ZhouX.2009. Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system. Blood, 114:1319–1330.
