Mitochondrial gene editing could add to debate

Researchers at the Salk Institute for Biological Studies have used gene editing to eliminate mutations in mitochondrial DNA in mice. The findings are likely to fuel the debate about germline gene editing, which has raised concerns about the ethics of altering DNA that is passed to future generations, even for heritable diseases for which no treatment is available (see BioCentury Innovations, March 26).

In the study, published in Cell, researchers used two techniques, involving either mitochondrially-targeted restriction nucleases or transcription activator-like effector nucleases (TALENs), to eliminate specific mitochondrial DNA sequences in mouse oocytes and, separately, in one-cell mouse embryos. The mitochondrial changes were transmitted to the first generation of mice. In addition, the Salk group used the technique in mammalian oocytes to edit mutations in human mitochondrial genes responsible for the mitochondrial diseases Leber's hereditary optic neuropathy and dystonia and neurogenic muscle weakness, ataxia and retinitis pigmentosa. ...