ArticleInternational journal of biological sciences2026
Alleviation of mutant TDP-43-mediated neuropathology by inducible stem cells in monkeys.
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32 authors.
Xichen SongState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Caijuan LiState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Yang YangGuangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, 510632, China.
Chunhui HuangState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Min ChenSouth China Institute of Large Animal Models for Biomedicine, Wuyi University, Jiangmen, 529000, China.
Song LinDepartment of Physiology School of Medicine, Jinan University, Guangzhou, 510632, China.
Zhonghai HuangState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Wei WangState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Kai LiaoThe First Affiliated Hospital of Jinan University, Guangzhou, 510632, China.
Huiyi WeiThe First Affiliated Hospital of Jinan University, Guangzhou, 510632, China.
Lu WangThe First Affiliated Hospital of Jinan University, Guangzhou, 510632, China.
Hao XuThe First Affiliated Hospital of Jinan University, Guangzhou, 510632, China.
Yizhi ChenState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Yingqi LinState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Jiawei LiState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Zhen DaiGuangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, 510632, China.
Wenguang XieGuangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, 510632, China.
Xiao ZhengState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Jianhao WuState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Jiale GaoState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Jiaxi WuState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Zhuchi TuState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Libing ZhouState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Lu HuangState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Chaoran RenState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Kwok-Fai SoState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Peng YinState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Huiming YangSun Yat-sen University, Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, Guangzhou, China.
Shihua LiState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Liangxue LaiGuangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, 510632, China.
Xiao-Jiang LiState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Sen YanState Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, Guangdong Provincial Key Laboratory of Non-human Primate Research, Guangdong-Hong Kong-Macau Institute of CNS Regeneration, School of Medicine, Jinan University, Guangzhou,510632, China.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Abnormal cytoplasmic accumulation of TAR DNA-binding protein 43 (TDP-43) is a common pathological feature of TDP-43 proteinopathies. Since non-human primate models can better recapitulate this neuropathology than rodents, we used a monkey model to evaluate the therapeutic potential of stem cells for TDP-43-mediated neuropathology. We established a cynomolgus monkey model by expressing mutant TDP-43 (M337V) in the monkey striatum through AAV injection. This model exhibited motor dysfunction and abnormal cytoplasmic TDP-43 accumulation. Using multi-gene modified stem cells (NILB-hiPSCs) that can be induced to differentiate
Indexed as
DNA-Binding ProteinsInduced Pluripotent Stem CellsTDP-43 ProteinopathiesAnimalsCell DifferentiationDisease Models, AnimalMacaca fascicularisMutationDNA-Binding Proteinscynomolgus monkeygenetically modified stem cellsNILB-hiPSCsTDP-43 proteinopathies
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