Mitochondrial Therapy for Disease Treatment and Prevention: What’s New?| Biomed Grid

Many diseases caused by mitochondrial dysfunction are caused by mitochondrial DNA abnormalities. The concept of mitochondrial therapy is based on the repair or removal of the existing anomaly. This can be done by replacing affected mitochondria by healthy ones, by in situ correction of the damaged mitochondrial DNA or by selective destruction of the affected mitochondria. In theory, all these concepts are applicable at the preconception stage, thus preventing the mother-to-child disease transmission, as well as at more advanced developmental stages (embryonic, fetal and postnatal) to alleviate disease symptoms in specific organs and tissues. Animal reasearch has paved the way to human therapies at all of these levels. So far, the only successful human application concerned the preconception stage and was based on replacement of diseased mitochondria in patients' oocytes with healthy ones originating from oocytes donated by healthy women. However, new developments in molecular biology techniques, enabling a direct reparation of mitochondrial DNA mutations or selective destruction of the affected mitocondria, will hopefully make it possible to treat mitochondrial diseases at both preconception and postconception stages, without the need for recourse to third-party (donor) biological material.