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Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene.
Int J Mol Sci. 2020 Jun 25;21(12):4511. doi: 10.3390/ijms21124511.
Int J Mol Sci. 2020.
PMID: 32630425
Free PMC article.
Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing.
McIntosh CS, Aung-Htut MT, Fletcher S, Wilton SD.
McIntosh CS, et al.
Int J Mol Sci. 2019 Oct 31;20(21):5434. doi: 10.3390/ijms20215434.
Int J Mol Sci. 2019.
PMID: 31683630
Free PMC article.
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