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...
| Format: | Article |
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| Language: | English |
| Published: |
Nature Portfolio
2023-02-01
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| Series: | Communications Medicine |
| Online Access: | https://doi.org/10.1038/s43856-023-00255-3 |