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Wang, Eric T

0000-0003-2655-5525

Mayo Clinic

Center for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.

Citations 1

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.