| Issue |
Med Sci (Paris)
Volume 37, Number 4, Avril 2021
|
|
|---|---|---|
| Page(s) | 413 - 416 | |
| Section | Chroniques génomiques | |
| DOI | https://doi.org/10.1051/medsci/2021054 | |
| Published online | 28 April 2021 | |
Vers une thérapie génique pour la progéria ?
Towards gene therapy for Progeria ?
UMR 7268 ADÉS, Aix-Marseille, Université /EFS/CNRS ; CoReBio PACA, case 901, Parc scientifique de Luminy, 13288 Marseille Cedex 09, France
Abstract
Hutchinson-Gilford Progeria (acute premature aging) is caused by a de novo point mutation in the lamin A gene. Recently, this mutation has been accurately corrected by base editing in patient cell lines and in a mouse model, resulting in nearly complete reversal to a normal phenotype. This success opens the perspective for clinical applications in Progeria and other diseases.
© 2021 médecine/sciences – Inserm
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