Mitochondria-targeted nanoparticles in treatment of neurodegenerative diseases

This review summarized the advantages and challenges of nanotechnology-based mitochondrial-targeted therapy for neurodegenerative diseases (NDs). In this review, relationships between mitochondrial dysfunction and NDs are discussed; the applications of various NPs in treatment and diagnosis of mitochondrial dysfunction related NDs are also introduced.


Neurodegenerative diseases (NDs) are a class of heterogeneous diseases that includes Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis. Mitochondria play an important role in oxidative balance and metabolic activity of neurons; therefore, mitochondrial dysfunction is associated with NDs and mitochondria are considered a potential treatment target for NDs. Several obstacles, including the blood-brain barrier (BBB) and cell/mitochondrial membranes, reduce the efficiency of drug entry into the target lesions. Therefore, a variety of neuron mitochondrial targeting strategies has been developed. Among them, nanotechnology-based treatments show especially promising results. Owing to their adjustable size, appropriate charge, and lipophilic surface, nanoparticles (NPs) are the ideal theranostic system for crossing the BBB and targeting the neuronal mitochondria. In this review, we discussed the role of dysfunctional mitochondria in ND pathogenesis as well as the physiological barriers to various treatment strategies. We also reviewed the use and advantages of various NPs (including organic, inorganic, and biological membrane-coated NPs) for the treatment and diagnosis of NDs. Finally, we summarized the evidence and possible use for the promising role of NP-based theranostic systems in the treatment of mitochondrial dysfunction-related NDs.

Author list:

Yue Zhang, Han Yang, Daohe Wei, Xinhui Zhang, Jian Wang, Xiaoli Wu*, Jin Chang*

How to cite:

Y. Zhang, H. Yang, D. Wei, X.Zhang, J. Wang, X. Wu, J. Chang, Exploration 2021, 1, 20210115.