Annual review of biochemistry

Improving How Precisely Gene-Editing Tools Target Specific Genes

Updated

Abstract

Programmable nucleases and deaminases may cause off-target activity, leading to potential genomic damage.

  • These gene-editing tools can modify genomes but may also recognize similar DNA sequences, causing unintended changes.
  • Off-target activity can result in local mutations or larger genomic rearrangements.
  • Measuring and limiting genome-wide off-target effects is crucial for safe therapeutic applications.
  • Various methods exist for identifying off-target sites in both cells and cell-free environments.
  • Strategies have been developed to reduce off-target activity in programmable gene-editing systems.

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