Genetic & Genomic MedicinemedRxivPreprint
Antisense oligonucleotide depletion of CCDC146 is a broad-spectrum therapeutic strategy for ALS
Sai Zhang, Tobias Moll, Jasper Rubin-Sigler…·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.
5 citations