Low-dose MyoAAV 2A-mediated delivery of engineered micro-utrophin achieves pan- muscle tissue distribution with elevated muscle function in Duchenne muscular dystrophy
Na Li, Zhuo Li, Zilian Zhang, Zhikai Xiahou, Hong Xiao, Shiwen Wu, Fuqiang Xu, Qing Liu, Yang Wu, Yafeng Song · 2026
Read Low-dose MyoAAV 2A-mediated delivery of engineered micro-utrophin achieves pan- muscle tissue distribution with elevated muscle function in Duchenne muscular dystrophy by Na Li, Zhuo Li, Zilian Zhang, Zhikai Xiahou, Hong Xiao, Shiwen Wu, Fuqiang Xu, Qing Liu, Yang Wu, Yafeng Song (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13321518.txt.
Description
Na Li and co-authors test a low dose gene therapy for Duchenne muscular dystrophy in a 2026 mouse study. The disease stems from missing dystrophin, a protein that shields muscle from damage. Gene therapy helps but often needs high virus doses that raise toxicity risks. The team used a muscle seeking virus shell to deliver a small engineered utrophin, a stand-in protein.
Newborn mdx mice got a single low dose, and tissues were checked four weeks later. The stand-in protein appeared across skeletal muscle, diaphragm, and heart, with little spill into the liver. Damage markers in blood and muscle fell, dye leakage dropped, and grip and treadmill performance improved, which supports low dose delivery.
Catalog record
| CREATOR | Na Li, Zhuo Li, Zilian Zhang, Zhikai Xiahou, Hong Xiao, Shiwen Wu, Fuqiang Xu, Qing Liu, Yang Wu, Yafeng Song |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 79146 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |