Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice
Background: Therapeutic activation of endogenous utrophin, a homolog of dystrophin, represents a mutation-agnostic therapeutic strategy with considerable potential for treating Duchenne muscular dystrophy (DMD). However, the large size of current dCas9-based activation systems hinders efficient aden...
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| Autori principali: | , , , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Elsevier
2025-12-01
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| Serie: | Regenerative Therapy |
| Soggetti: | |
| Accesso online: | http://www.sciencedirect.com/science/article/pii/S2352320425002081 |
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