| Issue |
Med Sci (Paris)
Volume 41, Number 11, Novembre 2025
Chroniques génomiques
|
|
|---|---|---|
| Page(s) | 935 - 937 | |
| Section | Forum | |
| DOI | https://doi.org/10.1051/medsci/2025220 | |
| Published online | 12 December 2025 | |
Vers une thérapie génique pour la maladie de Huntington
Towards gene therapy for Hutington’s disease
Biologiste, généticien, Président d’Aprogène (association pour la promotion de la génomique), Marseille, France
*
This email address is being protected from spambots. You need JavaScript enabled to view it.
Abstract
Building on recent discoveries on the pathogenic mechanism of Huntinton’s disease, the foundations of a gene therapy for this disease have been established : in vitro and in vivo base editing interrupting the CAG repeat region of the gene prevents the somatic amplification responsible for the pathology. Although much remains to be done, this opens the perspective of a gene therapy for this disease.
© 2025 médecine/sciences – Inserm
Article publié sous les conditions définies par la licence Creative Commons Attribution License CC-BY (https://creativecommons.org/licenses/by/4.0), qui autorise sans restrictions l’utilisation, la diffusion, et la reproduction sur quelque support que ce soit, sous réserve de citation correcte de la publication originale.
Current usage metrics show cumulative count of Article Views (full-text article views including HTML views, PDF and ePub downloads, according to the available data) and Abstracts Views on Vision4Press platform.
Data correspond to usage on the plateform after 2015. The current usage metrics is available 48-96 hours after online publication and is updated daily on week days.
Initial download of the metrics may take a while.