Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.
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Nat Med. 2022 Jan;28(1):117-124. doi: 10.1038/s41591-021-01557-6. Epub 2021 Dec 23.
Nat Med. 2022.
PMID: 34949835
Free PMC article.