Le oligonucléotides antisens Il s'agit de la première thérapie génique (en fait thérapie par ARN antisens, puisqu'ils ne modifient pas le code génétique) disponibles sur le marché pour le traitement des maladies neurogénétiques. Ils sont basés sur la technique de saut d'exon (Duchenne) ou inclusion d'exons (AME). Un traitement contre la dystrophie myotonique de Steinert sera également bientôt disponible.
- Nusinersen (Spinraza).
- Eteplirsen (Exondys51).
- Golodirsen (Vyondys 53).
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Daguenet E, Dujardin G, Valcárcel J. The pathogenicity of splicing defects: mechanistic insights into pre-mRNA processing inform novel therapeutic approaches. EMBO reports [Internet]. 2015 Dec [cited 2022 Nov 3];16(12):1640–55. Available from: https://www.embopress.org/doi/full/10.15252/embr.201541116