In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor
Christiano R. R. Alves, Sabyasachi Das, Vijai Krishnan, Leillani L. Ha, Lauren R. Fox, Hannah E. Stutzman, Claire E. Shamber, Pazhanichamy Kalailingam, Siobhan McCarthy, Christian Lacks Lino Cardenas, C.K. Fong, Takahiko Imai, Sunayana Mitra, Shuqi Yun, Rachael K. Wood, , 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.
Nature Biomedical Engineering · Sep 11, 2025Peer-reviewedBioengineering