A cyclic pyrrole-imidazole polyamide reduces pathogenic RNA in CAG/CTG triplet repeat neurological disease models.
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Ikenoshita S, et al. Among authors: sugiyama h.
J Clin Invest. 2023 Nov 15;133(22):e164792. doi: 10.1172/JCI164792.
J Clin Invest. 2023.
PMID: 37707954
Free PMC article.