| AAV | Adeno-associated virus |
| BBB | Blood–brain barrier |
| CNS | Central nervous system |
| CRISPR | Clustered Regularly Interspaced Short Palindromic Repeats |
| sgRNA | Single-guide RNA |
| MELAS | Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes |
| MERRE | Myoclonic Epilepsy with Ragged Red Fibers |
| mtDNA | Mitochondrial DNA |
| MTS | Mitochondria targeting signal |
| PAM | Protospacer adjacent motif |
| NARP | Neurogenic muscle weakness, ataxia, and retinitis pigmentosa |
| lncRNA | Long non-coding RNA |
| LHON | Leber hereditary optic neuropathy |
| TALEN | Transcription Activator-Like Effector Nucleases |
| rAAV | Recombinant Adeno-associated virus |
| RIC | RNA Import Complex |
| OxPhos | Oxidative phosphorylation |
| ZFN | Zinc-finger nucleases |
| UTR | Untranslated Regions |