Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation

Journal of Central Nervous System Disease

Review

Aino Vesikansa¹ 

[1] Department of Veterinary Biosciences, Faculty of Veterinary Medicine, University of Helsinki, Helsinki, Finland;Neuroscience Center, Helsinki Institute of Life Science, University of Helsinki, Helsinki, Finland.;

关键词: Central nervous system;    CRISPR-Cas;    CRISPR;    brain;    CNS disease;    genome editing;   

DOI  :  10.1177/1179573518787469

 received in 2018-01-09, accepted in 2018-06-09,  发布年份 2018

来源: Sage Journals

【 摘 要 】

The complex structure and highly variable gene expression profile of the brain makes it among the most challenging fields to study in both basic and translational biological research. Most of the brain diseases are multifactorial and despite the rapidly increasing genomic data, molecular pathways and causal links between genes and central nervous system (CNS) diseases are largely unknown. The advent of an easy and flexible CRISPR-Cas genome editing technology has rapidly revolutionized the field of functional genomics and opened unprecedented possibilities to dissect the mechanisms of CNS disease. CRISPR-Cas allows a plenitude of applications for both gene-focused and genome-wide approaches, ranging from original “gene scissors” making permanent modifications in the genome to the regulation of gene expression and epigenetics. CRISPR technology provides a unique opportunity to establish new cellular and animal models of CNS diseases and holds potential for breakthroughs in the CNS research and drug development.

【 授权许可】

CC BY-NC   
© The Author(s) 2018

【 图 表 】

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