Targeting mitochondrial function to treat optic neuropathy

Mitochondrion. 2017 Sep;36:7-14. doi: 10.1016/j.mito.2016.07.013. Epub 2016 Jul 28.


Many reports have illustrated a tight connection between vision and mitochondrial function. Not only are most mitochondrial diseases associated with some form of vision impairment, many ophthalmological disorders such as glaucoma, age-related macular degeneration and diabetic retinopathy also show signs of mitochondrial dysfunction. Despite a vast amount of evidence, vision loss is still only treated symptomatically, which is only partially a consequence of resistance to acknowledge that mitochondria could be the common denominator and hence a promising therapeutic target. More importantly, clinical support of this concept is only emerging. Moreover, only a few drug candidates and treatment strategies are in development or approved that selectively aim to restore mitochondrial function. This review rationalizes the currently developed therapeutic approaches that target mitochondrial function by discussing their proposed mode(s) of action and provides an overview on their development status with regards to optic neuropathies.

Keywords: Age-related macular degeneration; CoQ(10); Diabetic retinopathy; EPI-743; Glaucoma; Idebenone; Leber's hereditary optic neuropathy (LHON); MTP-131; MitoQ; Mitochondrial dysfunction; Ocluvia; Optic neuropathy; Retinopathy; SkQ1.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Mitochondria / drug effects*
  • Mitochondria / physiology*
  • Neuroprotective Agents / therapeutic use*
  • Optic Nerve Diseases / drug therapy*
  • Optic Nerve Diseases / physiopathology*


  • Neuroprotective Agents