Mitochondrial diseases are very heterogeneous in their genetic cause and clinical manifestation. During the last few decades progress has been made in the diagnosis of mitochondrial diseases, but an established therapy is so far lacking. Several experimental strategies targeting different points of intervention are currently being assessed world-wide. Numerous mouse models of OXPHOS disorders have become available enabling further optimization and validation of therapeutic strategies and paving the way for future clinical trials. In this review, we provide an update on current developments towards treatment as well as the potential and status of transition into therapeutic use.
Keywords: Experimental therapy; Mitochondrial disease; Mitochondrial medicine; Treatment.
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