Antisense oligonucleotide depletion of CCDC146 is a broad-spectrum therapeutic strategy for ALS
Sai Zhang, Tobias Moll, Jasper Rubin-Sigler, Sharon Tu, Shuya Li, Enming Yuan, Menghui Liu, Marriane King, Elham M. Alhathli, Connie Treanor, Alison E. Twelvetrees, Marcel Weinreich, Sam Bonsall, Calum Harvey, Sarah Gornall, , Michael P. Snyder
The paper identifies CCDC146 as a modifier of ALS survival through primary cilia dysfunction and shows ASO targeting of CCDC146 rescues ALS phenotypes in human and mouse models.
medRxiv · Mar 30, 2024 · PreprintGenetic & Genomic Medicine