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Published on: April 10, 2019
Mesenchymal stem cell-derived exosomes improve motor function and attenuate neuropathology in a mouse model of
Hua-Jing You1, Shu-Bin Fang2, Teng-Teng Wu1
1Department of Neurology, The First Affiliated Hospital, Sun Yat-sen University; Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, National Key Clinical Department and Key Discipline of Neurology, No. 58 Zhongshan Road 2, Guangzhou, 510080, Guangdong, China.
Background:
Machado-Joseph disease is the most common autosomal dominant hereditary ataxia worldwide without effective treatment. Mesenchymal stem cells (MSCs) could slow the disease progression, but side effects limited their clinical application. Besides, MSC-derived exosomes exerted similar efficacy and have many advantages over MSCs. The aim of this study was to examine the efficacy of MSC-derived exosomes in YACMJD84.2 mice.
Methods:
Rotarod performance was evaluated every 2 weeks after a presymptomatic administration of intravenous MSC-derived exosomes twice in YACMJD84.2 mice. Loss of Purkinje cells, relative expression level of Bcl-2/Bax, cerebellar myelin loss, and neuroinflammation were assessed 8 weeks following treatment.
Results:
MSC-derived exosomes were isolated and purified through anion exchange chromatography. Better coordination in rotarod performance was maintained for 6 weeks in YACMJD84.2 mice with exosomal treatment, compared with those without exosomal treatment. Neuropathological changes including loss of Purkinje cells, cerebellar myelin loss, and neuroinflammation were also attenuated 8 weeks after exosomal treatment. The higher relative ratio of Bcl-2/Bax was consistent with the attenuation of loss of Purkinje cells.
Conclusions:
MSC-derived exosomes could promote rotarod performance and attenuate neuropathology, including loss of Purkinje cells, cerebellar myelin loss, and neuroinflammation. Therefore, MSC-derived exosomes have a great potential in the treatment of Machado-Joseph disease.
Insights
Mesenchymal stem cell-derived exosomes show promise for treating Machado-Joseph disease by improving motor function and reducing brain damage in mice. This offers a potential new therapeutic avenue for this inherited ataxia.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Genetics
Background:
- Machado-Joseph disease is a prevalent, untreatable autosomal dominant hereditary ataxia.
- Mesenchymal stem cells (MSCs) show therapeutic potential but have limitations.
- MSC-derived exosomes offer similar efficacy with enhanced advantages for clinical application.
Purpose of the Study:
- To evaluate the therapeutic efficacy of MSC-derived exosomes in a mouse model of Machado-Joseph disease (YACMJD84.2 mice).
Main Methods:
- Presymptomatic intravenous administration of MSC-derived exosomes twice in YACMJD84.2 mice.
- Assessment of rotarod performance every two weeks.
- Evaluation of Purkinje cell loss, Bcl-2/Bax ratio, cerebellar myelin loss, and neuroinflammation eight weeks post-treatment.
Main Results:
- Exosome treatment improved rotarod performance for six weeks in treated mice.
- Neuropathological changes, including Purkinje cell loss, myelin loss, and neuroinflammation, were attenuated.
- A higher Bcl-2/Bax ratio correlated with reduced Purkinje cell loss.
Conclusions:
- MSC-derived exosomes significantly improved motor function and reduced neuropathology in a Machado-Joseph disease mouse model.
- These findings highlight the potential of MSC-derived exosomes as a viable treatment for Machado-Joseph disease.
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