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干细胞移植在亨廷顿舞蹈症中的发病机制及潜在治疗应用

Pathogenesis and potential therapeutic application of stem cells transplantation in Huntington's disease.

作者信息

Saha Sudeepto, Dey Manami Jayati, Promon Salman Khan, Araf Yusha

机构信息

Department of Life Sciences, School of Environment and Life Sciences, Independent University, Bangladesh (IUB), Dhaka, Bangladesh.

Department of Pharmaceutical Sciences, North South University, Dhaka, Bangladesh.

出版信息

Regen Ther. 2022 Sep 25;21:406-412. doi: 10.1016/j.reth.2022.09.001. eCollection 2022 Dec.

DOI:10.1016/j.reth.2022.09.001
PMID:36196447
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9513215/
Abstract

Huntington's disease (HD) is a progressive neurodegenerative disorder which is caused due to repetitive CAG or glutamine expression along the coding region of the Huntington gene. This disease results in certain movement abnormalities, affective disturbances, dementia and cognitive impairments. To this date, there is no proper cure for this rare and fatal neurological condition but there have been certain advancements in the field of genetic animal model research studies to elucidate the understanding of the pathogenesis of this condition. Currently, HD follows a certain therapeutic approach which just relieves the symptoms but doesn't cure the underlying cause of the disease. Stem cell therapy can be a breakthrough in developing a potential cure for this condition. In this review, we have discussed the pathogenesis and the efficacy and clinical practicality of the therapeutic application of stem cell transplantation in Huntington's disease. The application of this groundbreaking therapy on genetically altered animal models has been listed and analyzed in brief.

摘要

亨廷顿舞蹈症(HD)是一种进行性神经退行性疾病,由亨廷顿基因编码区域的CAG重复或谷氨酰胺表达所致。该疾病会导致特定的运动异常、情感障碍、痴呆和认知障碍。迄今为止,对于这种罕见且致命的神经疾病尚无有效的治愈方法,但在遗传动物模型研究领域已取得了一定进展,以阐明对该疾病发病机制的理解。目前,HD采用的某种治疗方法仅能缓解症状,无法治愈疾病的根本病因。干细胞疗法可能是开发针对该疾病潜在治愈方法的一个突破。在本综述中,我们讨论了干细胞移植治疗亨廷顿舞蹈症的发病机制、疗效及临床实用性。还简要列出并分析了这种开创性疗法在基因改造动物模型上的应用情况。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5e6c/9513215/7545a3d09b78/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5e6c/9513215/7545a3d09b78/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5e6c/9513215/7545a3d09b78/gr1.jpg

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J Control Release. 2021 Feb 10;330:1152-1167. doi: 10.1016/j.jconrel.2020.11.021. Epub 2020 Nov 13.
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Proteostasis in Huntington's disease: disease mechanisms and therapeutic opportunities.亨廷顿病中的蛋白稳态:疾病机制和治疗机会。
Acta Pharmacol Sin. 2018 May;39(5):754-769. doi: 10.1038/aps.2018.11. Epub 2018 Apr 5.
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Therapeutic effects of stem cells in rodent models of Huntington's disease: Review and electrophysiological findings.
干细胞在亨廷顿病啮齿动物模型中的治疗效果:综述和电生理发现。
CNS Neurosci Ther. 2018 Apr;24(4):329-342. doi: 10.1111/cns.12839. Epub 2018 Mar 6.
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Human Neural Stem Cell Transplantation Rescues Functional Deficits in R6/2 and Q140 Huntington's Disease Mice.人神经干细胞移植可挽救 R6/2 和 Q140 亨廷顿病模型鼠的功能缺陷。
Stem Cell Reports. 2018 Jan 9;10(1):58-72. doi: 10.1016/j.stemcr.2017.11.005. Epub 2017 Dec 7.
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Induced Pluripotent Stem Cell-Derived Neural Stem Cell Transplantations Reduced Behavioral Deficits and Ameliorated Neuropathological Changes in YAC128 Mouse Model of Huntington's Disease.诱导多能干细胞衍生的神经干细胞移植减轻了亨廷顿舞蹈病YAC128小鼠模型的行为缺陷并改善了神经病理变化。
Front Neurosci. 2017 Nov 10;11:628. doi: 10.3389/fnins.2017.00628. eCollection 2017.
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Concise Review: The Use of Stem Cells for Understanding and Treating Huntington's Disease.简明综述:干细胞在亨廷顿病的理解和治疗中的应用。
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