Cell models and drug discovery for mitochondrial diseases.

Author: Shuang-Yi HU ¹ ; Qian-Qian ZHUANG ¹ ; Yue QIU ¹ ; Xu-Fen ZHU ¹ ; Qing-Feng YAN ¹
Author Information

1. Institute of Genetics and Regenerative Biology, College of Life Sciences, Zhejiang University, Hangzhou 310058, China.
Publication Type:Journal Article
Keywords: Mitochondrial diseases; Mitochondrial DNA; Cell model; Drug discovery
From: Journal of Zhejiang University. Science. B 2019;20(5):449-456
CountryChina
Language:English
Abstract: Mitochondrion is a semi-autonomous organelle, important for cell energy metabolism, apoptosis, the production of reactive oxygen species (ROS), and Ca homeostasis. Mitochondrial DNA (mtDNA) mutation is one of the primary factors in mitochondrial disorders. Though much progress has been made, there remain many difficulties in constructing cell models for mitochondrial diseases. This seriously restricts studies related to targeted drug discovery and the mechanism and therapy for such diseases. Here we summarize the characteristics of patient-specific immortalized lymphoblastoid cells, fibroblastoid cells, cytoplasmic hybrid (cybrid) cell lines, and induced pluripotent stem cells (iPSCs)-derived differentiation cells in the study of mitochondrial disorders, as well as offering discussion of roles and advances of these cell models, particularly in the screening of drugs.