Introduction: Research has revealed a relationship between mitochondrial dysfunction and diseases such as diabetes, ischemia-reperfusion injury, cancer and many more. As a result, mitochondria have gained attention as a target organelle for the treatment of many diseases. Successful delivery of the drug molecule to the mitochondria could be achieved by keeping in mind the normal intracellular trafficking fate of molecules in cell as well as through the mitochondria and exploring the new possibilities to reach the target in an efficient manner.
Areas covered: This review covers important areas such as structure and physiology of mitochondria, mitochondrial genome and its role in the diseases led by mitochondrial dysfunction, generation of reactive oxygen species and its disbalance in pathophysiological conditions and apoptosis. Further, the review focuses on various human mitochondrial diseases, particularly cancer, and strategies and methods of targeting drug and genetic materials to mitochondria. Novel nanotechnology-based carriers for mitochondria delivery are discussed with an attempt of providing readers with a current and future prospective of mitochondrial therapeutics.
Expert opinion: Numerous investigators have attempted to establish a mitochondrial drug delivery system; still, many hurdles yet remain to be overcome before mitochondrial medicine reaches clinical applications. We need to develop a delivery system to encapsulate drugs, proteins and genes that would be practically viable for scale-up and strategies to target and regulate drug release to the cytosol after endosomal escape, and thereafter to deliver the released drug to the mitochondria. Current innovations in the nanotechnology could be effectively utilized with mitochondrial medicine for designing optimal nanoparticle drug delivery system for mitochondrial diseases on clinical setting.