Back to Authors

Chen, Jonathan L.

https://orcid.org/0000-0001-7907-9732

A5080126683 | I123431417

Scripps Research Institute

Citations 2

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.

Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD

ACS Chemical Biology | November 16, 2020 | Volume: 15 | Issue: 12 | Pages: 3112-3123

10.1021/acschembio.0c00049 | PMID 33196168