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Chemical and Biological Approaches to Interrogate off-Target Effects of Genome Editing Tools

Zhixin Lei1,2, Haowei Meng3, Yuan Zhuang4

  • 1Peking-Tsinghua Center for Life Sciences, Peking University, Beijing100871, China.

ACS Chemical Biology
|February 2, 2023
PubMed

Insights

Genome editing tools require careful assessment for unintended DNA changes, known as off-target effects, especially for therapeutic uses. This review covers these effects, detection methods, and alternative RNA editing tools for research and medicine.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Programmable genome editing tools enable precise DNA modification at specific sites.
  • Assessing off-target effects is critical for the safe application of genome editing technologies, particularly in therapeutics.
  • Unintended alterations in the genome can have significant biological and clinical consequences.

Purpose of the Study:

  • To review observed off-target effects associated with various genome editing tools.
  • To discuss methods for detecting and evaluating off-target genome editing.
  • To explore RNA editing tools as potential alternatives for therapeutic applications.

Main Methods:

  • Literature review of genome editing technologies and their off-target effects.
  • Analysis of current methods for detecting off-target mutations.
  • Summary of existing RNA editing tools and their applications in therapy.

Main Results:

  • Various genome editing tools exhibit distinct off-target profiles.
  • Multiple detection methods exist, each with specific advantages and limitations.
  • RNA editing tools offer promising alternative strategies for therapeutic interventions.

Conclusions:

  • Comprehensive evaluation of off-target effects is essential for safe genome editing.
  • Understanding detection method limitations is key to accurate assessment.
  • RNA editing presents a viable alternative for therapeutic applications in research and medicine.

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