Gene-editing in patient and humanized-mice primary muscle stem cells rescues dysferlin expression in dysferlin-deficient muscular dystrophy
| Authors | Escobar Helena, Di Francescantonio Silvia, Smirnova Julia, Graf Robin, Müthel Stefanie, Marg Andreas, Zhogov Alexej, Krishna Supriya, Metzler Eric, Petkova Mina, Daumke Oliver, Kühn Ralf, Spuler Simone |
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| Journal | Nature Communications |
| Publication Date | 2025-01-02 |
| DOI | 10.1038/s41467-024-55086-0 |