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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...
| Formato: | Online Artículo Texto |
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| Lenguaje: | English |
| Publicado: |
Nature Publishing Group UK
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9974939/ https://www.ncbi.nlm.nih.gov/pubmed/36854978 http://dx.doi.org/10.1038/s43856-023-00255-3 |