Oct 17, 2015

Advances in therapeutic CRISPR/Cas9 genome editing

Translational Research : the Journal of Laboratory and Clinical Medicine
Nataša Savić, Gerald Schwank

Abstract

Targeted nucleases are widely used as tools for genome editing. Two years ago the clustered regularly interspaced short palindromic repeat (CRISPR)-associated Cas9 nuclease was used for the first time, and since then has largely revolutionized the field. The tremendous success of the CRISPR/Cas9 genome editing tool is powered by the ease design principle of the guide RNA that targets Cas9 to the desired DNA locus, and by the high specificity and efficiency of CRISPR/Cas9-generated DNA breaks. Several studies recently used CRISPR/Cas9 to successfully modulate disease-causing alleles in vivo in animal models and ex vivo in somatic and induced pluripotent stem cells, raising hope for therapeutic genome editing in the clinics. In this review, we will summarize and discuss such preclinical CRISPR/Cas9 gene therapy reports.

  • References44
  • Citations25

Citations

Mentioned in this Paper

Embryo
Zinc Fingers
CRYGC
Study
In Vivo
Non-Homologous DNA End-Joining
T-Lymphocyte
DMD gene
Bacterial Proteins
Specimen Type - Fibroblasts

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