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Modulation of RNA Splicing by Oligonucleotides: Mechanisms of Action and Therapeutic Implications

14 Feb 2022

Abstract excerpt

Dysregulation of RNA splicing causes many diseases and disorders. Several therapeutic approaches have been developed to correct aberrant alternative splicing events for the treatment of cancers and hereditary diseases, including gene therapy and redirecting splicing, using small molecules or splice switching oligonucleotides (SSO). Significant advances in the chemistry and pharmacology of nucleic acid have led to...

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PMID: 35166605
DOI: 10.1089/nat.2021.0067

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