[Towards gene therapy for Progeria ?]
| Autor: | Jordan B; UMR 7268 ADÉS, Aix-Marseille, Université /EFS/CNRS ; CoReBio PACA, case 901, Parc scientifique de Luminy, 13288 Marseille Cedex 09, France. |
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| Jazyk: | francouzština |
| Zdroj: | Medecine sciences : M/S [Med Sci (Paris)] 2021 Apr; Vol. 37 (4), pp. 413-416. Date of Electronic Publication: 2021 Apr 28. |
| DOI: | 10.1051/medsci/2021054 |
| Abstrakt: | 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.) |
| Databáze: | MEDLINE |
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