NeuroscienceMolecular Therapy — Nucleic AcidsPeer-reviewed
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 B. Carroll
This paper demonstrates that antisense oligonucleotides targeting human ATN1 protect against DRPLA pathology in a fully humanized mouse model.
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