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| Item Type: | Article |
|---|---|
| Title: | Stammzelltherapien für Muskeldystrophien |
| Creators Name: | Escobar, H. |
| Abstract: | Muscular dystrophies are devastating and untreatable genetic diseases leading to progressive muscle degeneration and weakness. The expanding landscape of CRISPR-Cas-based genome editing tools allows the in situ repair of many disease-causing mutations in patient cells in an unprecedented manner. Here, I discuss recent advances and challenges for using gene edited muscle stem cells in autologous cell replacement therapies to treat muscular dystrophy. |
| Source: | BIOspektrum |
| ISSN: | 0947-0867 |
| Publisher: | Springer / Spektrum Akademischer Verlag |
| Volume: | 28 |
| Page Range: | 493–496 |
| Date: | September 2022 |
| Official Publication: | https://doi.org/10.1007/s12268-022-1807-5 |
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