Edward Rebar

Abstract Engineered nucleases have gained broad appeal for their ability to mediate highly efficient genome editing. However the specificity of these reagents remains a concern, especially for therapeutic applications, given the potential mutagenic consequences of off-target cleavage.

Two parallel studies show gene editing can target mutant mitochondria in adult mice, providing an alternative path for drug developers to the controversial approach of modifying embryos. In back-to-back papers published in Nature Medicine on Sept.