Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells
Rachele Rossi, Silvia Torelli, Marc Moore, Pierpaolo Ala, Jennifer Morgan, Jyoti Malhotra, Francesco Muntoni · 2024
Read Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells by Rachele Rossi, Silvia Torelli, Marc Moore, Pierpaolo Ala, Jennifer Morgan, Jyoti Malhotra, Francesco Muntoni (2024) 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/pmc-11606086.txt.
Description
Rachele Rossi and six co-authors probe how a drug called golodirsen works in muscle cells from patients with Duchenne muscular dystrophy, in a study from 2024. The drug skips a faulty gene block so the cell can make a shorter but useful protein.
In lab grown muscle tubes, the drug cut out only the target block, as hoped. It also eased a known flaw where gene messages fade along their length, a first show of that effect in human cells. Yet more skipping did not always mean more protein, and much message stayed in the cell core, which may cap gains.
Catalog record
| CREATOR | Rachele Rossi, Silvia Torelli, Marc Moore, Pierpaolo Ala, Jennifer Morgan, Jyoti Malhotra, Francesco Muntoni |
|---|---|
| YEAR | 2024 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 55 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |