Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G
4
C
2
) repeat expansion in vitro and in vivo ALS models
Science Translational Medicine | October 27, 2021 | Volume: 13 | Issue: 617 | Pages: No page available
10.1126/scitranslmed.abd5991
| PMID 34705518
Small-molecule recruitment of an endogenous nuclease rescues pathologies associated with c9ALS/FTD in vivo by targeting an RNA repeat expansion.