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RNA-Targeting Splicing Modifiers: Drug Development and Screening Assays

14 Apr 2021

Abstract excerpt

RNA splicing is an essential step in producing mature messenger RNA (mRNA) and other RNA species. Harnessing RNA splicing modifiers as a new pharmacological modality is promising for the treatment of diseases caused by aberrant splicing. This drug modality can be used for infectious diseases by disrupting the splicing of essential pathogenic genes. Several antisense oligonucleotide splicing modifiers were...

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PMID: 33919699
DOI: 10.3390/molecules26082263

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