| Autor: |
Eriya Kenjo, Hiroyuki Hozumi, Yukimasa Makita, Kumiko A. Iwabuchi, Naoko Fujimoto, Satoru Matsumoto, Maya Kimura, Yuichiro Amano, Masataka Ifuku, Youichi Naoe, Naoto Inukai, Akitsu Hotta |
| Jazyk: |
angličtina |
| Rok vydání: |
2021 |
| Předmět: |
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| Zdroj: |
Nature Communications, Vol 12, Iss 1, Pp 1-13 (2021) |
| Druh dokumentu: |
article |
| ISSN: |
2041-1723 |
| DOI: |
10.1038/s41467-021-26714-w |
| Popis: |
In vivo delivery of CRISPR-Cas9 holds promise for treating muscular dystrophy, however, AAV delivery is known to be immunogenic. Here, the authors show that LNP delivery of CRISPR-Cas9 enables repeated injections into skeletal muscle and leads to restored dystrophin expression in multiple muscle groups. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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