Der Antisense-Oligonukleotide Sie sind die erste Gentherapie (eigentlich). Antisense-RNA-Therapie, da sie den genetischen Code nicht verändern), die auf dem Markt zur Behandlung neurogenetischer Erkrankungen erhältlich sind. Sie basieren auf der Technik von Exon-Skipping (Duchenne) oder Exon-Einschluss (AME). In Kürze wird es auch eine Behandlung der myotonen Steinert-Dystrophie geben.
- Nusinersen (Spinraza).
- Eteplirsen (Exondys51).
- Golodirsen (Vyondys 53).
19955111
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