Vers une thérapie génique pour la progéria ?
Résumé
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.
Pour citer ce document
Jordan, Bertrand ; Vers une thérapie génique pour la progéria ?, Med Sci (Paris), Vol. 37, N° 4 ; p. 413-416 ; DOI : 10.1051/medsci/2021054