BioengineeringNature Biomedical EngineeringPeer-reviewed
In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor
Christiano R. R. Alves, Sabyasachi Das, Vijai Krishnan…·Patricia Leonor Musolino
This paper develops a customized CRISPR-Cas9 base editor to correct the ACTA2 R179H mutation in a mouse model of MSMDS, improving survival and rescuing vascular and neurological phenotypes.
13 citations