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Article

Rapid and scalable personalized ASO screening in patient-derived organoids.

Nature - 1 Feb 2025

Means John C, Martinez-Bengochea Anabel L, Louiselle Daniel A, Nemechek Jacqelyn M, Perry John M, Farrow Emily G, Pastinen Tomi, Younger Scott T

Abstract excerpt

Personalized antisense oligonucleotides (ASOs) have achieved positive results in the treatment of rare genetic disease1. As clinical sequencing technologies continue to advance, the ability to identify patients with rare disease harbouring pathogenic genetic variants amenable to this therapeutic strategy will probably improve. Here we describe a scalable platform for generating patient-derived cellular models and...

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Identifiers

PMID: 39843740
DOI: 10.1038/s41586-024-08462-1
PMCID: PMC11798851

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DMD

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