| Issue |
Med Sci (Paris)
Volume 40, Number 8-9, Août-Septembre 2024
Chroniques génomiques
|
|
|---|---|---|
| Page(s) | 677 - 679 | |
| Section | Forum | |
| DOI | https://doi.org/10.1051/medsci/2024093 | |
| Published online | 20 September 2024 | |
Thérapie génique in vivo : une approche modulaire
A modular approach to in vivo gene therapy
Biologiste, généticien et immunologiste, Président d’Aprogène (Association pour la promotion de la Génomique), 13007 Marseille, France
Abstract
In vivo inactivation of a deleterious gene has been achieved in a small trial, with excellent clinical results. Interestingly, the delivery and editing system is the same as in previous work on a different disease, and the new therapy required simply changing the guide RNA used to target the Cas9 nuclease. This modular approach could be extended to a number of other genetic diseases.
© 2024 médecine/sciences – Inserm
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