Mitochondrial transplantation as a promising therapy for mitochondrial diseases

Mitochondrial diseases are a group of inherited or acquired metabolic disorders caused by mitochondrial dysfunction which may affect almost all the organs in the body and present at any age. However, no satisfactory therapeutic strategies have been available for mitochondrial diseases so far. Mitochondrial transplantation is a burgeoning approach for treatment of mitochondrial diseases by recov-ery of dysfunctional mitochondria in defective cells using isolated functional mitochondria. Many models of mitochondrial transplantation in cells, animals, and patients have proved effective via various routes of mitochondrial delivery. This review presents different techniques used in mitochondrial isolation and de-livery, mechanisms of mitochondrial internalization and consequences of mitochondrial transplantation, along with challenges for clinical application. Despite some unknowns and challenges, mitochondrial transplantation would provide an innovative approach for mitochondrial medicine.(c) 2023 Chinese Pharmaceutical Association and Institute of Materia Medica, Chinese Academy of Medical Sciences. Production and hosting by Elsevier B.V. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

Automated summary

Mitochondrial diseases are metabolic disorders caused by mitochondrial dysfunction, which can affect any organ and occur at any age. Currently, there are no satisfactory therapeutic strategies for these diseases. Mitochondrial transplantation is a promising approach that involves the recovery of dysfunctional mitochondria using isolated functional mitochondria, and it has been proven effective in various models. This review provides an overview of different techniques for mitochondrial isolation and delivery, the mechanisms of mitochondrial internalization, the consequences of transplantation, and the challenges for clinical application. Despite some unknowns and challenges, mitochondrial transplantation offers an innovative approach for mitochondrial medicine.