TableĀ 1 Overview of the therapeutic strategies for primary mitochondrial diseases
General strategies
  • Wide applicability

  • Potentially cost-effective

  • Address common pathomechanisms

  • Off-target effects

  • Activation of mitochondrial biogenesis

  • Regulating execution pathways (apoptosis, autophagy, and fission/fusion)

  • Shaping mitochondrial cristae

  • Bypass of RC defects by using xenogenes

  • Use of dNTPs

Tailored strategies
  • Targeted for a single disease

  • Potentially highly effective

  • Limited to a single/few conditions

  • Expensive

  • AAV-mediated gene replacement

  • Selective elimination of mutant mtDNA by ZNF or TALE nucleases