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Juni 2018 / Mol Ther Nucleic Acids The antibiotic-free pFAR4 vector paired with the Sleeping Beauty transposon system mediates efficient transgene delivery in human cells M. Pastor S. Johnen N. Harmening M. Quiviger J. Pailloux M. Kropp P. Walter Z. Ivics Z. Izsvák G. Thumann D. Scherman C. Marie 07. Februar 2018 / Neuron Precisely timed nicotinic activation drives SST inhibition in neocortical circuits J. Urban-Ciecko J.S. Jouhanneau S.E. Myal J.F.A. Poulet A.L. Barth 04. April 2018 / Mol Ther Efficient non-viral gene delivery into human hematopoietic stem cells by minicircle Sleeping Beauty transposon vectors M. Holstein C. Mesa-Nuñez C. Miskey E. Almarza V. Poletti M. Schmeer E. Grueso J.C. Ordóñez Flores D. Kobelt W. Walther M.K. Aneja J, Geiger H.B. Bonig Z. Izsvák M. Schleef C. Rudolph F. Mavilio J.A. Bueren G. Guenechea Z. Ivics 01. Mai 2018 / Hum Gene Ther Efficient non-viral T cell engineering by Sleeping Beauty minicircles diminishing DNA toxicity and miRNAs silencing the endogenous TCR J. Clauss M. Obenaus C. Miskey Z. Ivics Z. Izsvák W. Uckert M. Bunse 16. März 2018 / Mol Ther Methods Clin Dev A combined in vivo HSC transduction/selection approach results in efficient and stable gene expression in peripheral blood cells in mice H. Wang M. Richter N. Psatha C. Li J. Kim J. Liu A. Ehrhardt S.K. Nilsson B. Cao D. Palmer P. Ng Z. Izsvák K.G. Haworth H.P. Kiem T. Papayannopoulou A. Lieber 01. März 2018 / J Thromb Haemost High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease I. Portier K. Vanhoorelbeke S. Verhenne I. Pareyn N. Vandeputte H. Deckmyn D.S. Goldenberg H.B. Samal M. Singh Z. Ivics Z. Izsvák S.F. De Meyer 18. April 2018 / Nat Commun Single synaptic inputs drive high-precision action potentials in parvalbumin expressing GABA-ergic cortical neurons in vivo J.S. Jouhanneau J. Kremkow J.F.A. Poulet 15. Juni 2018 / Mol Ther Methods Clin Dev Integrating HDAd5/35++ vectors as a new platform for HSC gene therapy of hemoglobinopathies C. Li N. Psatha H. Wang M. Singh H.B. Samal W. Zhang A. Ehrhardt Z. Izsvák T. Papayannopoulou A. Lieber 01. Juni 2018 / Mol Ther Nucleic Acids The antibiotic-free pFAR4 vector paired with the sleeping beauty transposon sSystem mediates efficient transgene delivery in human cells M. Pastor S. Johnen N. Harmening M. Quiviger J. Pailloux M. Kropp P. Walter Z. Ivics Z. Izsvák G. Thumann D. Scherman C. Marie 01. November 2018 / Eur J Med Genet Improved molecular platform for the gene therapy of rare diseases by liver protein secretion M. Quiviger A. Giannakopoulos S. Verhenne C. Marie E.F. Stavrou K. Vanhoorelbeke Z. Izsvák S.F. De Meyer A. Athanassiadou D. 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01. Juni 2018 / Mol Ther Nucleic Acids The antibiotic-free pFAR4 vector paired with the Sleeping Beauty transposon system mediates efficient transgene delivery in human cells M. Pastor S. Johnen N. Harmening M. Quiviger J. Pailloux M. Kropp P. Walter Z. Ivics Z. Izsvák G. Thumann D. Scherman C. Marie
07. Februar 2018 / Neuron Precisely timed nicotinic activation drives SST inhibition in neocortical circuits J. Urban-Ciecko J.S. Jouhanneau S.E. Myal J.F.A. Poulet A.L. Barth
04. April 2018 / Mol Ther Efficient non-viral gene delivery into human hematopoietic stem cells by minicircle Sleeping Beauty transposon vectors M. Holstein C. Mesa-Nuñez C. Miskey E. Almarza V. Poletti M. Schmeer E. Grueso J.C. Ordóñez Flores D. Kobelt W. Walther M.K. Aneja J, Geiger H.B. Bonig Z. Izsvák M. Schleef C. Rudolph F. Mavilio J.A. Bueren G. Guenechea Z. Ivics
01. Mai 2018 / Hum Gene Ther Efficient non-viral T cell engineering by Sleeping Beauty minicircles diminishing DNA toxicity and miRNAs silencing the endogenous TCR J. Clauss M. Obenaus C. Miskey Z. Ivics Z. Izsvák W. Uckert M. Bunse
16. März 2018 / Mol Ther Methods Clin Dev A combined in vivo HSC transduction/selection approach results in efficient and stable gene expression in peripheral blood cells in mice H. Wang M. Richter N. Psatha C. Li J. Kim J. Liu A. Ehrhardt S.K. Nilsson B. Cao D. Palmer P. Ng Z. Izsvák K.G. Haworth H.P. Kiem T. Papayannopoulou A. Lieber
01. März 2018 / J Thromb Haemost High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease I. Portier K. Vanhoorelbeke S. Verhenne I. Pareyn N. Vandeputte H. Deckmyn D.S. Goldenberg H.B. Samal M. Singh Z. Ivics Z. Izsvák S.F. De Meyer
18. April 2018 / Nat Commun Single synaptic inputs drive high-precision action potentials in parvalbumin expressing GABA-ergic cortical neurons in vivo J.S. Jouhanneau J. Kremkow J.F.A. Poulet
15. Juni 2018 / Mol Ther Methods Clin Dev Integrating HDAd5/35++ vectors as a new platform for HSC gene therapy of hemoglobinopathies C. Li N. Psatha H. Wang M. Singh H.B. Samal W. Zhang A. Ehrhardt Z. Izsvák T. Papayannopoulou A. Lieber
01. Juni 2018 / Mol Ther Nucleic Acids The antibiotic-free pFAR4 vector paired with the sleeping beauty transposon sSystem mediates efficient transgene delivery in human cells M. Pastor S. Johnen N. Harmening M. Quiviger J. Pailloux M. Kropp P. Walter Z. Ivics Z. Izsvák G. Thumann D. Scherman C. Marie
01. November 2018 / Eur J Med Genet Improved molecular platform for the gene therapy of rare diseases by liver protein secretion M. Quiviger A. Giannakopoulos S. Verhenne C. Marie E.F. Stavrou K. Vanhoorelbeke Z. Izsvák S.F. De Meyer A. Athanassiadou D. Scherman