Cartier-LacaveN., Hacein-Bey-AbinaS., BartholomaeC.C., et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science, 326, 818–823.
2.
ChuahM., MatesL., BelayE., et al. (2009). Molecular evolution of a novel hyperactive sleeping beauty transposase enables robust stable gene transfer in vertebrates. Nat. Genet., 41, 753–761.
3.
DreesenI.A., LüchingerN.A., StarkW.J., et al. (2009). Tricalcium phosphate nanoparticles enable rapid purification, increase transduction kinetics, and modify the tropism of mammalian viruses. Biotechnol. Bioeng., 102, 1197–1207.
4.
GabrielR., EckenbergR., ParuzynskiA., et al. (2009). Comprehensive genomic access to vector integration in clinical gene therapy. Nat. Med., 15, 1431–1436.
5.
AnlikerB., AbelT., KneisslS., et al. (2010). Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors. Nat. Methods, 7, 929–935.
6.
BelayE., MátraiJ., Acosta-SanchezA., et al. (2010). Novel hyperactive transposons for genetic modification of induced pluripotent and adult stem cells: a nonviral paradigm for coaxed differentiation. Stem Cells, 28, 1760–1771.
7.
BoztugK., SchmidtM., SchwarzerA., et al. (2010). Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N. Engl. J. Med., 363, 1918–1927.
8.
BusskampV., DuebelJ., BalyaD., et al. (2010). Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science, 329, 413–417.
9.
RoweH.M., JakobssonJ., MesnardD., et al. (2010). KAP1 controls endogenous retroviruses in embryonic stem cells. Nature, 463, 237–240.
10.
SteinS., OttM.G., Schultze-StrasserS., et al. (2010). Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med., 16, 198–204.
11.
TronoD., Van LintC., RouziouxC., et al. (2010). HIV persistence and the prospect of long-term drug-free remissions for HIV-infected individuals. Science, 329, 174–180.
12.
Di StefanoB., MaffiolettiS.M., GentnerB., et al. (2011). A microRNA-based system for selecting and maintaining the pluripotent state in human induced pluripotent stem cells. Stem Cells, 29, 1684–1695.
13.
GabrielR., LombardoA., ArensA., et al. (2011). An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat. Biotechnol., 29, 816–823.
14.
LombardoA., CesanaD., GenoveseP., et al. (2011). Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat. Methods, 8, 861–869.
15.
MiharadaK., KarlssonG., RehnM., et al. (2011). Cripto regulates hematopoietic stem cells as a hypoxic-niche-related factor through cell surface receptor GRP78. Cell Stem Cell, 9, 330–344.
16.
YeH., Daoud-El BabaM., PengR.W., et al. (2011). A synthetic optogenetic transcription device enhances blood-glucose homeostasis in mice. Science, 332, 1565–1568.
LechmanE.R., GentnerB., van GalenP., et al. (2012). Attenuation of miR-126 activity expands HSC in vivo without exhaustion. Cell Stem Cell, 11, 799–811.
19.
AiutiA., BiascoL., ScaramuzzaS., et al. (2013). Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science, 341, 1233151.
20.
BardeI., RauwelB., Marin-FlorezR.M., et al. (2013). A KRAB/KAP1-miRNA cascade regulates erythropoiesis through stage-specific control of mitophagy. Science, 340, 350–353.
