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A Broad Application of CRISPR Cas9 in Infectious, Inflammatory and Neurodegenerative Diseases.
Journal of Neuroimmune Pharmacology ( IF 6.2 ) Pub Date : 2019-11-28 , DOI: 10.1007/s11481-019-09889-4
Kalipada Pahan 1, 2
Affiliation  

Being the most important immune-responsive cell type of the CNS, microglia always glorify the so-called crossroad of Neurology, Immunology and Pharmacology. As microglial activation is a hallmark of different neurodegenerative disorders including Alzheimer’s disease (AD), Parkinson’s disease (PD), HIV-associated neurocognitive disorders (HAND), Amyotrophic lateral sclerosis (ALS), etc., selective targeting of microglial cell signaling may be a valid option to control these neurodegenerative disorders with lesser side effects. This is particularly important as no effective therapies are available against these diseases and available neuroimmune modulators are known to target multiple cell types in a non-cell-specific manner. How we can achieve such specificity? A newly-developed cutting-edge molecular biology tool is rocking biomedical research in recent years so much so that it has already come under major lawsuits between the University of California Berkeley and the MIT-Harvard Broad Institute regarding its ownership rights, probably halting the Nobel committee to announce the most coveted prize to its owners. It is none other than Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR). In nutshell, the Cas9 enzyme has been paired with the bacterial immune system, CRISPR, to ultimately turn CRISPR/Cas9 as an effective genome editor. Therefore, this special issue has been devoted to highlight some of the recent discoveries on CRISPR/Cas9 in neurodegenerative disorders and explain these discoveries in the light of neuroimmune pharmacology.

中文翻译:

CRISPR Cas9在传染性,炎性和神经退行性疾病中的广泛应用。

小胶质细胞是中枢神经系统最重要的免疫应答细胞类型,它总是美化所谓的神经病学,免疫学和药理学的十字路口。由于小胶质细胞激活是不同神经退行性疾病的标志,包括阿尔茨海默氏病(AD),帕金森氏病(PD),HIV相关神经认知障碍(HAND),肌萎缩性侧索硬化症(ALS)等,因此可能是针对小胶质细胞信号传导的选择性靶点控制这些神经退行性疾病副作用较小的有效选择。这是特别重要的,因为没有针对这些疾病的有效疗法,并且已知可用的神经免疫调节剂以非细胞特异性方式靶向多种细胞类型。我们如何实现这种特异性?近年来,一种新开发的尖端分子生物学工具正在极大地影响生物医学研究,以至于它已经受到加州大学伯克利分校和麻省理工学院-哈佛大学广泛研究所之间关于其所有权的重大诉讼,可能使诺贝尔奖停止了委员会向其所有者宣布最令人垂涎​​的奖项。它就是簇状规则间隔的短回文重复序列(CRISPR)。简而言之,Cas9酶已与细菌免疫系统CRISPR配对,最终使CRISPR / Cas9成为有效的基因组编辑器。因此,本期专刊着重介绍了CRISPR / Cas9在神经退行性疾病中的一些最新发现,并根据神经免疫药理学来解释这些发现。
更新日期:2019-11-28
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