Mutation-agnostic base editing of the progerin farnesylation site rescues Hutchinson-Gilford progeria syndrome phenotypes in neuromuscular organoids
Dong-Woo Kim, Eun-Ji Kwon, Beom-Jin Jeon, Dong-Hyeok Kwon, Goo Jang, Youngyoon Yoon, Yuna Hwang, Hyukjin Lee, Hyuk-Jin Cha · 2026
Read Mutation-agnostic base editing of the progerin farnesylation site rescues Hutchinson-Gilford progeria syndrome phenotypes in neuromuscular organoids by Dong-Woo Kim, Eun-Ji Kwon, Beom-Jin Jeon, Dong-Hyeok Kwon, Goo Jang, Youngyoon Yoon, Yuna Hwang, Hyukjin Lee, Hyuk-Jin Cha (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/pmc-13627717.txt.
Description
Dong-Woo Kim and co-authors edit a shared defect site to rescue progeria traits in lab grown tissue in a 2026 study. The syndrome ages children fast and kills early, driven by a faulty, fat tagged form of a nuclear protein. Current drugs give modest gains, and edits aimed at one mutation miss atypical cases.
Their platform, FATE, rewrites the protein's fat tag site so the harmful tag cannot attach, whatever the patient's exact mutation. In stem cell grown nerve and muscle organoids, the faulty protein pooled in muscle, trapped a repair factor, and blocked damage repair. Editing cleared the pools, freed the factor, and restored repair and normal cell architecture.
Catalog record
| CREATOR | Dong-Woo Kim, Eun-Ji Kwon, Beom-Jin Jeon, Dong-Hyeok Kwon, Goo Jang, Youngyoon Yoon, Yuna Hwang, Hyukjin Lee, Hyuk-Jin Cha |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 82 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |