Molecular therapy : the journal of the American Society of Gene Therapy

Gene editing in living body cells using base and prime techniques

Updated

Abstract

Base editors and prime editors can effectively deliver precise DNA changes in animal models.

  • Base editors can correct the four most common single-base substitutions without causing extensive unwanted changes.
  • Prime editors can install any substitution, insertion, or deletion over a range of dozens of base pairs.
  • These editing technologies show greater efficiency and fewer byproducts compared to traditional methods that create double-strand DNA breaks.
  • Both viral and non-viral methods have been used to successfully deliver these genome editing tools in vivo.
  • The review highlights examples of somatic cell editing and discusses future development prospects.

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