Article

Systematic analysis of genetic and phenotypic characteristics reveals antisense oligonucleotide therapy potential for one-third of neurodevelopmental disorders

2025-03-20

Abstract excerpt

<h4>Background</h4> Neurodevelopmental disorders (NDDs) are a challenging group of disorders to treat, but promising therapeutic interventions in the form of antisense oligonucleotides (AONs) have emerged in recent years. However, the applicability of AON therapy for NDDs varies based on genetic and phenotypic traits. In this study we systematically evaluated key characteristics for AON therapy suitability in NDD...

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Literature Corpus work: 04a8ed2c-2c36-5561-8d30-28fb865301ee
DOI: 10.1101/2025.03.20.644369

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