Author

Leslie G. Biesecker

2 papers indexed here

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Open access Sep 2026

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...

Shaima Raji Abdul Rahiman Sirajuddeen, M. Lindhurst, Megan Detels et al. · Genetics in Medicine · 0 citations

#gene editing Open access Sep 2026

In Vivo Base Editing Partially Rescues Bone Dysplasia in a Mouse Model of Hutchinson‐Gilford Progeria Syndrome

In vivo delivery of a locus‐specific DNA base editor to bone tissue is demonstrated, the timing of treatment required for maximum efficacy is delineated, and it is suggested that this system might be tailored for application to other monogenic bone disorders.

Wayne A. Cabral, Caleb M. Grenko, Diana Yeritsyan et al. · Aging Cell · 0 citations