Background: Mitochondria are intracellular organelles involved in energy production, which play important roles in metabolism. Consequently, mutation in mitochondrial DNA may have adverse effects on the host organism. This hypothesis is supported by increasing number of reports that associate various diseases with the mutation of the mitochondrial genome. Therefore, new therapy methods for targeting mitochondria genome should be developed for the treatment of these diseases.
Objectives: The current progress in mitochondrial targeting gene delivery is discussed and future direction is suggested.
Methods: Recent research progress in this field is briefly introduced, and successes and obstacles in research are discussed.
Results/conclusion: Delivery of antisense DNA using lipophilic cation showed possible therapeutic effect in vitro. Delivery of tRNA is also another possible approach to correct tRNA mutations. However, research into the delivery of protein expression system using liposome and polymer has been very limited. The results suggest that more research is required to address the problems in mitochondrial targeting gene delivery. Here, we suggest 'multifunctional multilamella vesicular or multifunctional multi-vesicular (MMV)' for efficient mitochondrial targeting DNA delivery.