Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice
Collectively, these preclinical findings establish MyoAAV-saRNA as a promising and translatable platform for utrophin induction, highlighting its therapeutic potential for DMD.
Authors
- Zhikai Xiahou
- Tianyuan Wang (ORCID: https://orcid.org/0000-0002-6976-3626)
- Zilian Zhang
- Xinwei Hou (ORCID: https://orcid.org/0000-0002-7534-1569)
- Hong Xiao (ORCID: https://orcid.org/0000-0002-5113-2367)
- Yanming Gan
- Na Li (ORCID: https://orcid.org/0000-0002-4112-0944)
- Zhuo Li (ORCID: https://orcid.org/0000-0002-8288-3841)
- Yafeng Song
Institutions
- Hainan Normal University (CN)
- Qinghai Normal University (CN)
- Xi'an Medical University (CN)
- China Institute of Sport Science (CN)
- Beijing Sport University (CN)
- Northeastern University (CN)
Publication Details
- Journal
- Regenerative Therapy
- Published
- 2025-11-05
- DOI
- https://doi.org/10.1016/j.reth.2025.10.011
- Citations
- 1
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81