State Key Laboratory of Membrane Biology, Institute of Molecular Medicine, College of Future Technology, Peking University, China. (Q.H., J.L., F.L., L.J., P.X., W ...

Duke Congenital Heart Surgery Research and Training Laboratory, Duke University, Durham, NC (N.K.P., B.A., M.M.-M., D.M.O., J.W.T.). Department of Surgery, Duke ...

View all available purchase options and get full access to this article.

Methods: To develop a comprehensive therapy for MSMDS, we utilized CRISPR-Cas9 adenine base editing to correct the ACTA2 R179H mutation. We generated isogenic human induced pluripotent stem cell (iPSC ...