Intrathecal (G4C2)149 delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarks
Katelyn A Russell, Amelia A Shahrabi, Suleyman C Akerman, Matthew D Byrne, Jeffrey D Rothstein, Davide Trotti, Brigid K Jensen, Aaron R Haeusler · 2026
Read Intrathecal (G4C2)149 delivery in C9orf72-deficient mice yields mild motor dysfunction and ALS/FTD pathological hallmarks by Katelyn A Russell, Amelia A Shahrabi, Suleyman C Akerman, Matthew D Byrne, Jeffrey D Rothstein, Davide Trotti, Brigid K Jensen, Aaron R Haeusler (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/PMC13476920.txt.
Description
Katelyn A Russell and co-authors deliver a repeat DNA stretch in 2026 into mice lacking a gene tied to Lou Gehrig disease and dementia. The repeat in C9orf72 is the most common gene cause of both diseases. A virus carrier injected at birth spread the repeat widely, with strong reach into the spinal cord. Mice carried graded loss of their own gene copy to test combined effects.
Repeat carriers grew mildly weaker over time, while coordination faults tracked gene loss more than repeats. Spinal zones showed repeat protein buildup, motor cell loss, and immune cell activation. Sparse marks of the hallmark protein fault also appeared, plus faulty message splicing. The mild model suits early-stage drug tests.
Catalog record
| CREATOR | Katelyn A Russell, Amelia A Shahrabi, Suleyman C Akerman, Matthew D Byrne, Jeffrey D Rothstein, Davide Trotti, Brigid K Jensen, Aaron R Haeusler |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 87205 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |