Atrophin-1 Antisense Oligonucleotide Provides Robust Protection from Pathology in a Fully Humanized DRPLA Model
Velvet L. Smith, Bereket Z. Gidi, Robert M. Bragg, Jeffrey P. Cantle, Aliza Ben-Varon, Briana Noble, Sílvia Prades, Andrea Compton, Julie Greenfield, Joanna A. Korecka, Anya Gemos, Timothy W. Yu, Vikram Khurana, Holly B. Kordasiewicz, Hien Tran Zhao, , Jeffrey B. Carroll
This paper demonstrates that antisense oligonucleotides targeting human ATN1 protect against DRPLA pathology in a fully humanized mouse model.
Molecular Therapy — Nucleic Acids · Dec 31, 2025Peer-reviewedNeuroscience