CRISPR-Cas systems: Overview, innovations and applications in human disease research and gene therapy

Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations....

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Published inComputational and structural biotechnology journal Vol. 18; pp. 2401 - 2415
Main Authors Xu, Yuanyuan, Li, Zhanjun
Format Journal Article
LanguageEnglish
Published Elsevier B.V 01.01.2020
Research Network of Computational and Structural Biotechnology
Elsevier
Subjects
biotechnology
Cas9
CRISPR
CRISPR-Cas systems
DNA
Gene therapy
genes
Genome editing
Human disease models
human diseases
molecular biology
Off target effects
Rabbit
Review
CRISPR
Off target effects
Rabbit
Genome editing
Human disease models
Cas9
Gene therapy
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Abstract Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.
AbstractList Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.
Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.
Author Xu, Yuanyuan
Li, Zhanjun
Author_xml – sequence: 1
  givenname: Yuanyuan
  surname: Xu
  fullname: Xu, Yuanyuan
– sequence: 2
  givenname: Zhanjun
  surname: Li
  fullname: Li, Zhanjun
  email: lizj_1998@jlu.edu.cn
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Off target effects
Rabbit
Genome editing
Human disease models
Cas9
Gene therapy
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Snippet Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and...
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SubjectTerms biotechnology
Cas9
CRISPR
CRISPR-Cas systems
DNA
Gene therapy
genes
Genome editing
Human disease models
human diseases
molecular biology
Off target effects
Rabbit
Review
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Title CRISPR-Cas systems: Overview, innovations and applications in human disease research and gene therapy
URI https://dx.doi.org/10.1016/j.csbj.2020.08.031
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