Targeting Staufen 1 with antisense oligonucleotides for treating ALS and SCA2
Daniel R. Scoles, Sharan Paul, Warunee Dansithong, Karla Patricia Figueroa, Mandi Gandelman, Feliks Royzen, Collin J. Anderson, Stefan M. Pulst
This paper develops antisense oligonucleotides targeting STAU1 to treat ALS and SCA2, showing efficacy in reducing STAU1 expression and improving disease markers in mouse models.
bioRxiv · Nov 16, 2022 · PreprintNeuroscience