Table 3.

Non-chemical clinical treatments for mitochondrial diseases.

Treatment	Mechanisms	Target disease	Clinical trials
LUMEVOQ (GS010, rAAV2/2-ND4),	MT-ND4 deficiency rescue via allotopic expression of normal MT-ND4 using recombinant AAV [170,171,172]	LHON (Leber hereditary optic neuropathy)	Phase 3 (Active) /NCT03293524
NR082 (rAAV2-ND4),			Phase 2/3 (Active) /NCT03153293
ScAAV2-P1ND4v2	MT-ND4 deficiency rescue via allotopic expression of normal MT-ND4 using self-complementary AAV [173]		Phase 1 (Active) /NCT02161380
Mitochondrial augmentation	Replacement of dysfunctional mitochondria with healthy-exogenous donor mitochondria using in vitro uptake [174]	mtDNA depletion disease (Pearson syndrome)	Phase 1/2 (Active) /NCT03384420
Mesoangioblasts (MABs)	Intra-arterial injection of in vitro cultured patient-autologous mesoangioblasts, which harbor far fewer mtDNA mutations despite a much higher mutation load in patient [175,176]	Mitochondrial myopathy with m.A3243G mutation	Phase 2 (Active) /NCT05962333