Article

Specificity and exon target space of splicing modifying compounds

2025-09-01

Abstract excerpt

<title>Abstract</title> <p>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 wea...

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Literature Corpus work: aca8a866-e5a2-52f6-b847-fa1b19db36be
DOI: 10.21203/rs.3.rs-7397910/v1

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