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Individualized ASO therapy for rare diseases

Annemieke Aartsma-Rus is a Professor of Translational Genetics at the Leiden University Medical Center. She was one of the pioneers of antisense oligonucleotide (ASO)-mediated exon skipping therapy for Duchenne muscular dystrophy (DMD). Her work focuses on the personalized approach to delivering exo...

Ausführliche Beschreibung

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Bibliografische Detailangaben
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Portfolio 2023-02-01
Schriftenreihe:Communications Medicine
Online-Zugang:https://doi.org/10.1038/s43856-023-00255-3
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