Specificity and exon target space of splicing modifying compounds

Abstract excerpt

Modulation of splicing has become an established therapeutic strategy, with proven clinical applications and continued potential to target specific exons to influence gene expression. Recent advancements led to the identification of small molecule splicing modifiers such as Risdiplam and Branaplam. These compounds induce the inclusion of exons that are typically skipped due to their weak 5′ splice site. While Risd...

Topics

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• Mutation
• Neurogenetic and Muscular Disorders Research
• Niacinamide
• Ribonucleoproteins, Small Nuclear
• RNA modifications and cancer
• RNA Research and Splicing
• RNA Splice Sites
• Viral Infections and Immunology Research

Identifiers and source

Literature Corpus work

c229aa6a-0d1a-5813-a009-584d327e3f47

DOI

10.1101/2025.08.14.670273