Research demonstrates that in vivo base editing effectively rescues liver pathophysiology and peroxisome dysfunction in a mouse model of Zellweger spectrum disorder. This breakthrough could pave the way for novel therapeutic strategies targeting this rare genetic condition.
in vivo base editing rescues liver pathophysiology and peroxisome dysfunction in a mouse model of zellweger spectrum disorder
Original title: “In vivo base editing rescues liver pathophysiology and peroxisome dysfunction in a mouse model of Zellweger spectrum disorder.”