Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation

Aino Vesikansa

Tutkimustuotos: ArtikkelijulkaisuKatsausartikkeliTieteellinenvertaisarvioitu

Kuvaus

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.
Alkuperäiskielienglanti
Artikkeli1179573518787469
LehtiJournal of central nervous system disease
Vuosikerta10
Sivut1-13
Sivumäärä13
ISSN1179-5735
DOI - pysyväislinkit
TilaJulkaistu - 10 heinäkuuta 2018
OKM-julkaisutyyppiA2 Katsausartikkeli tieteellisessä aikakauslehdessä

Tieteenalat

  • 3124 Neurologia ja psykiatria

Lainaa tätä

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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.",
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Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation. / Vesikansa, Aino.

julkaisussa: Journal of central nervous system disease, Vuosikerta 10, 1179573518787469, 10.07.2018, s. 1-13.

Tutkimustuotos: ArtikkelijulkaisuKatsausartikkeliTieteellinenvertaisarvioitu

TY - JOUR

T1 - Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation

AU - Vesikansa, Aino

PY - 2018/7/10

Y1 - 2018/7/10

N2 - 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.

AB - 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.

KW - Central nervous system

KW - CRISPR-Cas

KW - CRISPR

KW - brain

KW - CNS disease

KW - genome editing

KW - PLURIPOTENT STEM-CELLS

KW - STAPHYLOCOCCUS-AUREUS CAS9

KW - RNA-GUIDED ENDONUCLEASE

KW - IN-VIVO

KW - ANIMAL-MODELS

KW - HOMOLOGOUS RECOMBINATION

KW - HUNTINGTONS-DISEASE

KW - DNA-REPAIR

KW - NEURODEGENERATIVE DISEASES

KW - GERMLINE TRANSMISSION

KW - 3124 Neurology and psychiatry

U2 - 10.1177/1179573518787469

DO - 10.1177/1179573518787469

M3 - Review Article

VL - 10

SP - 1

EP - 13

JO - Journal of central nervous system disease

JF - Journal of central nervous system disease

SN - 1179-5735

M1 - 1179573518787469

ER -