Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.
Nat Commun. 2021 Feb 8;12(1):847. doi: 10.1038/s41467-021-21112-8.
Nat Commun. 2021.
PMID: 33558503
Free PMC article.