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Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G 4 C 2 ) repeat expansion in vitro and in vivo ALS models
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Science Translational Medicine | October 27, 2021 | Volume: 13 | Issue: 617 | Pages: No page available
Small-molecule recruitment of an endogenous nuclease rescues pathologies associated with c9ALS/FTD in vivo by targeting an RNA repeat expansion.
A Small Molecule Exploits Hidden Structural Features within the RNA Repeat Expansion That Causes c9ALS/FTD and Rescues Pathological Hallmarks
Ursu, Andrei , Baisden, Jared T. , Bush, Jessica A. , Taghavi, Amirhossein , Zhang, Yong‐Jie , Petrucelli, Leonard , Yildirim, Ilyas , Disney, Matthew D. , Yildirim, Ilyas , Disney, Matthew D , Zhang, Yong-Jie , Choudhary, Shruti , Gendron, Tania F.
ACS Chemical Neuroscience | October 22, 2021 | Volume: 12 | Issue: 21 | Pages: 4076-4089
Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD
Ursu, Andrei , Wang, Kye Won , Bush, Jessica A. , Chen, Jonathan L. , Baisden, Jared T. , Zhang, Yong‐Jie , Petrucelli, Leonard , Yildirim, Ilyas , Chen, Jonathan L , Gendron, Tania F. , Yildirim, Ilyas , Disney, Matthew D , Zhang, Yong-Jie , Choudhary, Shruti , Disney, Matthew D.
ACS Chemical Biology | November 16, 2020 | Volume: 15 | Issue: 12 | Pages: 3112-3123