Browse by Journal Title

2023

Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation.
Müthel, S. and Marg, A. and Ignak, B. and Kieshauer, J. and Escobar, H. and Stadelmann, C. and Spuler, S.
Molecular Therapy - Nucleic Acids 13 : 494-511. March 2023

2022

Efficient CRISPR/Cas9-mediated mutagenesis in primary human B cells for identifying plasma cell regulators.
Le, T.A. and Chu, V.T. and Lino, A.C. and Schrezenmeier, E. and Kressler, C. and Hamo, D. and Rajewsky, K. and Dörner, T. and Dang, V.D.
Molecular Therapy - Nucleic Acids 30 : 621-632. 13 December 2022

mRNA-mediated delivery of gene editing tools to human primary muscle stem cells.
Stadelmann, C. and Di Francescantonio, S. and Marg, A. and Müthel, S. and Spuler, S. and Escobar, H.
Molecular Therapy - Nucleic Acids 28 : 47-57. 14 June 2022

2018

Exon skipping in a Dysf-missense mutant mouse model.
Malcher, J. and Heidt, L. and Goyenvalle, A. and Escobar, H. and Marg, A. and Beley, C. and Benchaouir, R. and Bader, M. and Spuler, S. and García, L. and Schöwel, V.
Molecular Therapy - Nucleic Acids 13 : 198-207. 7 December 2018

The antibiotic-free pFAR4 vector paired with the sleeping beauty transposon sSystem mediates efficient transgene delivery in human cells.
Pastor, M. and Johnen, S. and Harmening, N. and Quiviger, M. and Pailloux, J. and Kropp, M. and Walter, P. and Ivics, Z. and Izsvák, Z. and Thumann, G. and Scherman, D. and Marie, C.
Molecular Therapy - Nucleic Acids 11 : 57-67. 1 June 2018

2017

Long-term PEDF release in rat iris and retinal epithelial cells after Sleeping Beauty transposon-mediated gene delivery.
Garcia-Garcia, L. and Recalde, S. and Hernandez, M. and Bezunartea, J. and Rodriguez-Madoz, J.R. and Johnen, S. and Diarra, S. and Marie, C. and Izsvák, Z. and Ivics, Z. and Scherman, D. and Kropp, M. and Thumann, G. and Prosper, F. and Fernandez-Robredo, P. and Garcia-Layana, A.
Molecular Therapy - Nucleic Acids 9 : 1-11. 15 December 2017

Engineering of PEDF-expressing primary pigment epithelial cells by the SB transposon system delivered by pFAR4 plasmids.
Thumann, G. and Harmening, N. and Prat-Souteyrand, C. and Marie, C. and Pastor, M. and Sebe, A. and Miskey, C. and Hurst, L. and Diarra, S. and Kropp, M. and Walter, P. and Scherman, D. and Ivics, Z. and Izsvák, Z. and Johnen, S.
Molecular Therapy - Nucleic Acids 6 : 302-314. 17 March 2017

2016

Full-length dysferlin transfer by the hyperactive Sleeping Beauty transposase restores dysferlin-deficient muscle.
Escobar, H. and Schöwel, V. and Spuler, S. and Marg, A. and Izsvák, Z.
Molecular Therapy - Nucleic Acids 5 : e277. 19 January 2016