Vers une thérapie génique pour la maladie de Huntington

Abstract

Building on recent discoveries on the pathogenic mechanism of Huntinton’s disease, the foundations of a gene therapy for this disease have been established : in vitro and in vivo base editing interrupting the CAG repeat region of the gene prevents the somatic amplification responsible for the pathology. Although much remains to be done, this opens the perspective of a gene therapy for this disease.