AAV-vector based gene therapy for mitochondrial disease: progress and future perspectives

Hanaford, A.R., Cho, YJ. & Nakai, H.; Orphanet J Rare Dis 17, 217 (2022). doi:10.1186/s13023-022-02324-7 

This review summarizes the preclinical findings of AAV vector-based gene replacement therapy for mitochondrial diseases including Friedreich ataxia, Leigh syndrome, Barth syndrome, ethylmalonic encephalopathy, and others.