Mitochondrial gene therapy shows potential application in Leber’s hereditary optic neuropathy (LHON)

While gene therapy research has made enormous progress in recent years targeting a broad range of dominant and recessive disorders, relatively less attention has been focused on gene disorders associated with the mitochondrial genome. Targeting highly differentiated neuronal cells has been more than challenging and so the prospect of specifically targeting a sub-cellular organelle has seemed only a remote possibility at best. However, this may change in the light of recently published research showing that gene delivery to the mitochondrion is not only possible but demonstrable in the case of findings that show the suppression of visual loss and optic atrophy in an experimental model of Leber’s hereditary optic neuropathy (LHON). Such encouraging results may be expected to have important implications not only for LHON, but also for a broad range of human disorders brought about through mitochondrial gene defects.