| Human satellite cells have regenerative capacity and are genetically manipulable A Marg, H Escobar, S Gloy, M Kufeld, J Zacher, A Spuler, C Birchmeier, ... The Journal of clinical investigation 124 (10), 4257-4265, 2014 | 92 | 2014 |
| Full-length dysferlin transfer by the hyperactive sleeping beauty transposase restores dysferlin-deficient muscle H Escobar, V Schöwel, S Spuler, A Marg, Z Izsvák Molecular Therapy-Nucleic Acids 5, 2016 | 41 | 2016 |
| Human muscle-derived CLEC14A-positive cells regenerate muscle independent of PAX7 A Marg, H Escobar, N Karaiskos, SA Grunwald, E Metzler, J Kieshauer, ... Nature communications 10 (1), 5776, 2019 | 33 | 2019 |
| Exon skipping in a Dysf-missense mutant mouse model J Malcher, L Heidt, A Goyenvalle, H Escobar, A Marg, C Beley, ... Molecular Therapy-Nucleic Acids 13, 198-207, 2018 | 20 | 2018 |
| Base editing repairs an SGCA mutation in human primary muscle stem cells H Escobar, A Krause, S Keiper, J Kieshauer, S Müthel, MG de Paredes, ... JCI insight 6 (10), 2021 | 18 | 2021 |
| mRNA-mediated delivery of gene editing tools to human primary muscle stem cells C Stadelmann, S Di Francescantonio, A Marg, S Müthel, S Spuler, ... Molecular Therapy-Nucleic Acids 28, 47-57, 2022 | 16 | 2022 |
| Generation of two human induced pluripotent stem cell lines derived from myoblasts (MDCi014-A) and from peripheral blood mononuclear cells (MDCi014-B) from the same donor E Metzler, N Telugu, S Diecke, S Spuler, H Escobar Stem Cell Research 48, 101998, 2020 | 7 | 2020 |
| Generation of three age and gender matched pairs of human induced pluripotent stem cells derived from myoblasts (MDCi011-A, MDCi012-A, MDCi013-A) and from peripheral blood … E Metzler, N Telugu, S Diecke, S Spuler, H Escobar Stem Cell Research 48, 101987, 2020 | 4 | 2020 |
| Localized irradiation of mouse legs using an image-guided robotic linear accelerator M Kufeld, H Escobar, A Marg, D Pasemann, V Budach, S Spuler Annals of translational medicine 5 (7), 2017 | 3 | 2017 |
| Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation S Müthel, A Marg, B Ignak, J Kieshauer, H Escobar, C Stadelmann, ... Molecular Therapy-Nucleic Acids 31, 494-511, 2023 | 2 | 2023 |
| LMNA Co-Regulated Gene Expression as a Suitable Readout after Precise Gene Correction H Wang, A Krause, H Escobar, S Müthel, E Metzler, S Spuler International Journal of Molecular Sciences 23 (24), 15525, 2022 | 1 | 2022 |
| Generation of hiPSC-derived skeletal muscle cells: exploiting the potential of skeletal muscle-derived hiPSCs E Metzler, H Escobar, DY Sunaga-Franze, S Sauer, S Diecke, S Spuler Biomedicines 10 (5), 1204, 2022 | 1 | 2022 |
| Gene-edited primary muscle stem cells rescue dysferlin-deficient muscular dystrophy H Escobar, S di Francescantonio, A Marg, A Zhogov, S Krishna, E Metzler, ... | | 2024 |
| Disintegration of the NuRD complex in primary human muscle stem cells in critical illness myopathy J Schneider, D Sundaravinayagam, A Blume, A Marg, S Grunwald, ... International Journal of Molecular Sciences 24 (3), 2772, 2023 | | 2023 |
| mRNA-Based Gene Editing in Primary Human Muscle Stem Cells C Stadelmann, S Di Francescantonio, H Escobar, A Marg, S Muethel, ... MOLECULAR THERAPY 30 (4), 588-588, 2022 | | 2022 |
| CRISPR-Cas9 Induced Re-Framing Repairs a Muscular Dystrophy-Causing DYSF Founder Mutation in Primary Patient Muscle Stem Cells and a Novel Humanized Mouse Model H Escobar, S Di Francescantonio, A Zhogov, E Metzler, A Krause, A Marg, ... MOLECULAR THERAPY 30 (4), 496-496, 2022 | | 2022 |
| GENE EDITING AND MOLECULAR THERAPY: P. 285Effective gene editing of the human DYSF founder mutation c. 1624delG in patient-derived cells and generation of analogous" humanized … HE Fernandez, A Zhogov, E Metzler, R Kühn, S Spuler Neuromuscular Disorders 29, S150, 2019 | | 2019 |
| O. 19PAX7 deficiency causes mild congenital myopathy with rigid spine and respiratory insufficiency H Amthor, A Marg, H Escobar, S Grunwald, E Metzler, J Kieshauer, ... Neuromuscular Disorders 29, S120, 2019 | | 2019 |
| Allele-Specific Gene Correction Using CRISPR-Cas9 in Compound Heterozygote SGCAmutations S Spuler, H Escobar, S Bashir, R Kuehn MOLECULAR THERAPY 27 (4), 49-49, 2019 | | 2019 |
| Precise gene editing of muscular dystrophy-causing mutations in patient-derived iPSCs H Escobar, E Metzler, D Rossa, S Spuler HUMAN GENE THERAPY 28 (12), A56-A56, 2017 | | 2017 |
