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Splice-switching antisense oligonucleotides for pediatric neurological disorders

25 Jul 2024

Abstract excerpt

Pediatric neurological disorders are frequently devastating and present unmet needs for effective medicine. The successful treatment of spinal muscular atrophy with splice-switching antisense oligonucleotides (SSO) indicates a feasible path to targeting neurological disorders by redirecting pre-mRNA splicing. One direct outcome is the development of SSOs to treat haploinsufficient disorders by targeting naturally...

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PMID: 39119251
DOI: 10.3389/fnmol.2024.1412964

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