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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. Shorrock HK, et al. Among authors: cleary jd. bioRxiv [Preprint]. 2024 Aug 17:2024.08.17.608349. doi: 10.1101/2024.08.17.608349. bioRxiv. 2024. PMID: 39211226 Free PMC article. Preprint.
124 results