Base editing: a novel cure for severe combined immunodeficiency

摘要

In a recent Cell article,McAuley,Kohn et al.showcased proof-of-principle of a promising gene therapy(GT)approach to correct the underlying genetic defect responsible for CD3δ-deficient severe combined immunodeficiency(SCID)using a CRISPR-Cas9-derived adenine-base-editor(ABE).1 This ground-breaking and important study could impact future GTs for monogenic diseases by providing novel tailored therapeutic options(Fig.1).