Towards gene therapy for Progeria ? [unknown]
Vers une thérapie génique pour la progéria ?

Bertrand Jordan
2021 M S.Médecine Sciences  
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.
doi:10.1051/medsci/2021054 pmid:33908864 fatcat:yepfiytntrfjxfhgravowmsdum