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.
Gespeichert in:
| Hauptverfasser: | , , , |
|---|---|
| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
13 September 2024
|
| In: |
Nature medicine
Year: 2024, Jahrgang: 30, Heft: 10, Pages: 2725-2726 |
| ISSN: | 1546-170X |
| DOI: | 10.1038/s41591-024-03217-x |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/s41591-024-03217-x Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/s41591-024-03217-x 
|
| Verfasserangaben: | Rebecca Schuele, Matthis Synofzik, Holm Graessner & Annemieke Aartsma-Rus |
| Zusammenfassung: | 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. |
|---|---|
| Beschreibung: | Gesehen am 10.04.2025 |
| Beschreibung: | Online Resource |
| ISSN: | 1546-170X |
| DOI: | 10.1038/s41591-024-03217-x |