Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation

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Vesikansa , A 2018 , ' Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation ' , Journal of central nervous system disease , vol. 10 , 1179573518787469 , pp. 1-13 . https://doi.org/10.1177/1179573518787469

Title: Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation
Author: Vesikansa, Aino
Contributor: University of Helsinki, Veterinary Biosciences
Date: 2018-07-10
Language: eng
Number of pages: 13
Belongs to series: Journal of central nervous system disease
ISSN: 1179-5735
URI: http://hdl.handle.net/10138/299403
Abstract: 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.
Subject: Central nervous system
CRISPR-Cas
CRISPR
brain
CNS disease
genome editing
PLURIPOTENT STEM-CELLS
STAPHYLOCOCCUS-AUREUS CAS9
RNA-GUIDED ENDONUCLEASE
IN-VIVO
ANIMAL-MODELS
HOMOLOGOUS RECOMBINATION
HUNTINGTONS-DISEASE
DNA-REPAIR
NEURODEGENERATIVE DISEASES
GERMLINE TRANSMISSION
3124 Neurology and psychiatry
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