Rescue of Proteus syndrome lethality in mice with prenatal miransertib treatment.
PURPOSE Proteus syndrome is caused by constitutive AKT1 activation, resulting in severe overgrowth and tumor susceptibility. We developed a mouse model, featuring endogenously regulated, mosaic expression of the Akt1 c.49G>A, p.(Glu17Lys) (Akt1 E17K) variant. Prior results showed that ubiquitous Akt1E17K expression cau...