Genome Editing and Its Applications in Model Organisms

Genomics Proteomics Bioinformatics. 2015 Dec;13(6):336-44. doi: 10.1016/j.gpb.2015.12.001. Epub 2016 Jan 4.

Abstract

Technological advances are important for innovative biological research. Development of molecular tools for DNA manipulation, such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly-interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas), has revolutionized genome editing. These approaches can be used to develop potential therapeutic strategies to effectively treat heritable diseases. In the last few years, substantial progress has been made in CRISPR/Cas technology, including technical improvements and wide application in many model systems. This review describes recent advancements in genome editing with a particular focus on CRISPR/Cas, covering the underlying principles, technological optimization, and its application in zebrafish and other model organisms, disease modeling, and gene therapy used for personalized medicine.

Keywords: CRISPR/Cas; Disease model; Gene therapy; Genome editing; Zebrafish.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics
  • Deoxyribonucleases / genetics
  • Deoxyribonucleases / metabolism
  • Genetic Engineering / methods*
  • Genetic Engineering / statistics & numerical data*
  • Genetic Therapy / methods
  • Genome
  • Humans
  • Models, Biological
  • Mutagenesis / physiology
  • Zinc Fingers / genetics

Substances

  • Deoxyribonucleases