CAG repeat-selective compounds reduce abundance of expanded CAG RNAs in patient cell and murine models of SCAs.
Shorrock HK, Aliyeva A, Frias JA, DeMeo VA, Lennon CD, DeMeo CC, Mascorro AK, Shaughnessy S, Mazdiyasni H, Cleary JD, Reddy K, Vangaveti S, Shin DS, Berglund JA.
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bioRxiv [Preprint]. 2024 Aug 17:2024.08.17.608349. doi: 10.1101/2024.08.17.608349.
bioRxiv. 2024.
PMID: 39211226
Free PMC article.
Preprint.