Table 2 Experimental therapies for mitochondrial diseases
Targeted pathwayCompoundsReferences
Nucleotide metabolism• dCMP or tetrahydrouridine (inhibitor of cytidine deaminase)[9]
• dCMP +  dTMP[10]
PGC1α-dependent mitochondrial biogenesis• AICAR (via AMPK)[14]
• Bezafibrate (via PPARs)[18]
• NR (via Sirt1) or PARP inhibitors[13]
Mitochondrial shaping• Increasing L-Opa1[21]
• Inhibition of Oma1[23]
• SS peptides[24]
Bypassing OxPhos defects• Ndi1 (bypass for cI defects)[26,27]
• AOX (bypass for cIII/cIV defects)[28,29]
Shifting heteroplasmy• Restriction endonucleases[38,39]
• ZNF nucleases[40]
• TALE nucleases[41]
Elimination of toxic compounds• AAV-mediated gene therapy[34,36]
• Liver transplant[35]