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. La pathogénicité des défauts d'épissage : des connaissances mécanistiques sur le traitement des pré-ARNm éclairent de nouvelles approches thérapeutiques. Rapports EMBO [Internet]. Décembre 2015 [cité le 3 novembre 2022];16(12):1640-55. Disponible à partir de : https://www.embopress.org/doi/full/10.15252/embr.201541116