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Expansion of Splice-Switching Therapy with Antisense Oligonucleotides

4 Mar 2025

Abstract excerpt

Since 2016, splice-switching therapy, in which splicing is controlled by antisense oligonucleotides, has been applied in clinical practice for spinal muscular atrophy and Duchenne muscular dystrophy. In the former disease, this therapy induces exon inclusion, while, in the latter, it induces exon skipping, leading expression of functional proteins. Basic and clinical studies of splice-switching therapy for many...

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PMID: 40076889
DOI: 10.3390/ijms26052270

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