Activation of AMPK as a therapeutic strategy for FBXL4-related mitochondrial DNA depletion syndrome
Aniketh Bishnu, Juan Zapata-Muñoz, Robert W Taylor, Kei Sakamoto, Ian G Ganley · 2026
Read Activation of AMPK as a therapeutic strategy for FBXL4-related mitochondrial DNA depletion syndrome by Aniketh Bishnu, Juan Zapata-Muñoz, Robert W Taylor, Kei Sakamoto, Ian G Ganley (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/PMC13470155.txt.
Description
Aniketh Bishnu and colleagues test in 2026 whether switching on a cellular energy sensor can treat a rare mitochondrial disease. In the syndrome called MTDPS13, faulty FBXL4 lets two cleanup tags pile up, so cells destroy healthy mitochondria and energy output collapses. Children with the condition often die early, and no effective treatment exists.
The energy sensor AMPK was already known to hold back one cleanup route by sheltering a partner protein. Working in cells lacking FBXL4, in patient skin cells, and in a chemically induced mouse model, the team showed that drug activation of AMPK blocked the destructive cleanup. Functional mitochondrial content recovered as a result. The authors present AMPK activation as a realistic treatment strategy worth pursuing.
Catalog record
| CREATOR | Aniketh Bishnu, Juan Zapata-Muñoz, Robert W Taylor, Kei Sakamoto, Ian G Ganley |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 88448 |
| LICENSE | https://creativecommons.org/publicdomain/zero/1.0 |
| RIGHTS | CC0 |
| SOURCE | Europe PMC |