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Advancing ASO therapies from development to implementation

A novel application of antisense oligonucleotide (ASO) technology, developed to treat a single patient, adds to the growing number of ‘personalized’ therapies for rare diseases; but pathways to implementation and access are urgently needed.

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Bibliographic Details
Main Authors: Schüle-Freyer, Rebecca (Author) , Synofzik, Matthis (Author) , Graessner, Holm (Author) , Aartsma-Rus, Annemieke (Author)
Format: Article (Journal)
Language:English
Published: 13 September 2024
In: Nature medicine
Year: 2024, Volume: 30, Issue: 10, Pages: 2725-2726
ISSN:1546-170X
DOI:10.1038/s41591-024-03217-x
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/s41591-024-03217-x
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/s41591-024-03217-x
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Author Notes:Rebecca Schuele, Matthis Synofzik, Holm Graessner & Annemieke Aartsma-Rus
Description
Summary:A novel application of antisense oligonucleotide (ASO) technology, developed to treat a single patient, adds to the growing number of ‘personalized’ therapies for rare diseases; but pathways to implementation and access are urgently needed.
Item Description:Gesehen am 10.04.2025
Physical Description:Online Resource
ISSN:1546-170X
DOI:10.1038/s41591-024-03217-x

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