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Clustered regularly interspaced short palindromic repeats as an advanced treatment for Parkinson's disease
Brain and Behavior ( IF 3.1 ) Pub Date : 2021-07-21 , DOI: 10.1002/brb3.2280
Arshad Mehmood 1, 2 , Wajid Ali 3 , Zaheer Ud Din 4 , Shuang Song 1, 2 , Muhammad Sohail 5 , Wahid Shah 6 , Jiangyuan Guo 1, 2 , Ruo-Yi Guo 1, 2 , Ikram Ilahi 7 , Suleman Shah 8 , Fadhl Al-Shaebi 9 , Liaqat Zeb 10 , Ernest Amponsah Asiamah 11 , Zaid Al-Dhamin 12 , Hazrat Bilal 13 , Bin Li 1, 2
Affiliation  

Recently, genome-editing technology like clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 has improved the translational gap in the treatments mediated through gene therapy. The advantages of the CRISPR system, such as, work in the living cells and tissues, candidate this technique for the employing in experiments and the therapy of central nervous system diseases. Parkinson's disease (PD) is a widespread, disabling, neurodegenerative disease induced by dopaminergic neuron loss and linked to progressive motor impairment. Pathophysiological basis knowledge of PD has modified the PD classification model and expresses in the sporadic and familial types. Analyses of the earliest genetic linkage have shown in PD the inclusion of synuclein alpha (SNCA) genomic duplication and SNCA mutations in the familial types of PD pathogenesis. This review analyzes the structure, development, and function in genome editing regulated through the CRISPR/Cas9. Also, it explains the genes associated with PD pathogenesis and the appropriate modifications to favor PD. This study follows the direction by understanding the PD linking analyses in which the CRISPR technique is applied. Finally, this study explains the limitations and future trends of CRISPR service in relation to the genome-editing process in PD patients' induced pluripotent stem cells.

中文翻译:

成簇的规则间隔短回文重复作为帕金森病的高级治疗

最近,基因组编辑技术,如成簇的规则间隔短回文重复序列 (CRISPR)/Cas9,改善了通过基因治疗介导的治疗中的转化差距。CRISPR 系统的优势,例如在活细胞和组织中起作用,使该技术可用于实验和中枢神经系统疾病的治疗。帕金森病 (PD) 是一种由多巴胺能神经元丢失引起的广泛的、致残的神经退行性疾病,并与进行性运动障碍有关。PD的病理生理基础知识修改了PD分类模型,并在散发型和家族型中表达。对最早的遗传连锁的分析表明,在 PD 中,在家族性 PD 发病机制中包含了突触核蛋白 α (SNCA) 基因组重复和 SNCA 突变。本综述分析了通过 CRISPR/Cas9 调控的基因组编辑的结构、发展和功能。此外,它还解释了与 PD 发病机制相关的基因以及有利于 PD 的适当修饰。本研究通过了解应用 CRISPR 技术的 PD 链接分析来遵循方向。最后,本研究解释了 CRISPR 服务在 PD 患者诱导多能干细胞基因组编辑过程中的局限性和未来趋势。它解释了与 PD 发病机制相关的基因以及有利于 PD 的适当修饰。本研究通过了解应用 CRISPR 技术的 PD 链接分析来遵循方向。最后,本研究解释了 CRISPR 服务在 PD 患者诱导多能干细胞基因组编辑过程中的局限性和未来趋势。它解释了与 PD 发病机制相关的基因以及有利于 PD 的适当修饰。本研究通过了解应用 CRISPR 技术的 PD 链接分析来遵循方向。最后,本研究解释了 CRISPR 服务在 PD 患者诱导多能干细胞基因组编辑过程中的局限性和未来趋势。
更新日期:2021-09-03
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