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