2023

Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation.
Müthel, S. ORCID: https://orcid.org/0000-0002-3525-8859, Marg, A. ORCID: https://orcid.org/0000-0003-3673-4244, Ignak, B., Kieshauer, J. ORCID: https://orcid.org/0000-0002-6924-8616, Escobar, H. ORCID: https://orcid.org/0000-0002-8128-7845, Stadelmann, C. ORCID: https://orcid.org/0000-0001-5457-5721 and Spuler, S. ORCID: https://orcid.org/0000-0002-0155-1117
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., Chu, V.T. ORCID: https://orcid.org/0000-0003-1327-8696, Lino, A.C., Schrezenmeier, E., Kressler, C., Hamo, D., Rajewsky, K. ORCID: https://orcid.org/0000-0002-6633-6370, 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. ORCID: https://orcid.org/0000-0001-5457-5721, Di Francescantonio, S. ORCID: https://orcid.org/0000-0002-4815-491X, Marg, A. ORCID: https://orcid.org/0000-0003-3673-4244, Müthel, S. ORCID: https://orcid.org/0000-0002-3525-8859, Spuler, S. ORCID: https://orcid.org/0000-0002-0155-1117 and Escobar, H. ORCID: https://orcid.org/0000-0002-8128-7845
Molecular Therapy - Nucleic Acids 28 : 47-57. 14 June 2022

2018

Exon skipping in a Dysf-missense mutant mouse model.
Malcher, J., Heidt, L., Goyenvalle, A., Escobar, H. ORCID: https://orcid.org/0000-0002-8128-7845, Marg, A. ORCID: https://orcid.org/0000-0003-3673-4244, Beley, C., Benchaouir, R., Bader, M. ORCID: https://orcid.org/0000-0003-4780-4164, Spuler, S. ORCID: https://orcid.org/0000-0002-0155-1117, García, L. and Schöwel, V. ORCID: https://orcid.org/0000-0002-5924-3088
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., Johnen, S., Harmening, N., Quiviger, M., Pailloux, J., Kropp, M., Walter, P., Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658, Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384, Thumann, G., 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., Recalde, S., Hernandez, M., Bezunartea, J., Rodriguez-Madoz, J.R., Johnen, S., Diarra, S., Marie, C., Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384, Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658, Scherman, D., Kropp, M., Thumann, G., Prosper, F., 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., Harmening, N., Prat-Souteyrand, C., Marie, C., Pastor, M., Sebe, A., Miskey, C. ORCID: https://orcid.org/0000-0002-7566-9556, Hurst, L., Diarra, S., Kropp, M., Walter, P., Scherman, D., Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658, Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384 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. ORCID: https://orcid.org/0000-0002-8128-7845, Schöwel, V., Spuler, S. ORCID: https://orcid.org/0000-0002-0155-1117, Marg, A. ORCID: https://orcid.org/0000-0003-3673-4244 and Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384
Molecular Therapy - Nucleic Acids 5 : e277. 19 January 2016