BioCentury
ARTICLE | Targets & Mechanisms

Adult path for mitochondrial gene editing

How the possibility of mitochondrial gene editing in adults could accelerate translation

October 12, 2018 1:51 AM UTC

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. 24, teams from the University of Cambridge and the University of Miami demonstrated that gene editing by zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) can target mitochondrial genes in vivo in adult mice, raising the prospect the techniques could be used to treat a subset of mitochondrial diseases. ...