Duchenne muscular dystrophy: from gene to gene-ius therapies
Antoine Muchir, Antonio Musaro, Jeffrey Chamberlain · 2026
Read Duchenne muscular dystrophy: from gene to gene-ius therapies by Antoine Muchir, Antonio Musaro, Jeffrey Chamberlain (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/PMC13548582.txt.
Description
Antoine Muchir, Antonio Musaro, and Jeffrey Chamberlain review Duchenne muscular dystrophy from its gene defect to the newest genetic treatments, in a 2026 article. The disorder, passed on the X chromosome, removes the protein dystrophin, so muscles break down through childhood toward loss of walking, breathing failure, heart disease, and early death. Supportive care has improved, yet no cure exists, and its single-gene cause makes it a prime target for precision therapy.
The review explains the disease's mechanisms and natural course. It then walks through treatments that skip faulty gene sections, read through stop signals, or edit the genome directly. A further family delivers a shortened dystrophin gene by virus. Each approach faces limits, from working only for certain mutations to immune reactions and unknown durability. The authors see combination treatments and better delivery as the road ahead.
Catalog record
| CREATOR | Antoine Muchir, Antonio Musaro, Jeffrey Chamberlain |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 69 KB |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |