CLIP-Seq Analysis Enables the Design of Ribosomal RNA Bait Oligonucleotides That Protect Against C9orf72 ALS/FTD-Associated Poly-GR Pathophysiology
Juan Alberto Ortega, Ivan R. Sasselli, Marco Boccitto, Andrew C. Fleming, Tyler R. Fortuna, Yichen Li, Kohei Sato, Tristan D. Clemons, Elizabeth L. Daley, Thao Phuong Nguyen, Eric Nathaniel Anderson, Justin K. Ichida, Udai Bhan Pandey, Sandra L. Wolin, Samuel Isaac Stupp, Evangelos Kiskinis
The paper identifies that poly-GR binds ribosomal RNA and shows that modified rRNA oligonucleotides can mitigate poly-GR toxicity in models of C9orf72 ALS/FTD.
bioRxiv · Dec 30, 2022 · PreprintNeuroscience