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Article

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

Genome medicine - 21 May 2025

Wijnant Kim N, Nadif Kasri Nael, Vissers Lisenka E L M

Abstract excerpt

BACKGROUND: 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 NDDs, to...

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Genetic Therapy
Genetics and Neurodevelopmental Disorders
Genomics and Rare Diseases
Neurodevelopmental Disorders
Neurogenetic and Muscular Disorders Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Oligonucleotides, Antisense

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PMID: 40400017
DOI: 10.1186/s13073-025-01477-x
PMCID: PMC12096787

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