Expanding the Toolbox and Targets for Gene Editing

Trends Mol Med. 2021 Mar;27(3):203-206. doi: 10.1016/j.molmed.2020.12.005. Epub 2021 Jan 21.


Genome editing holds great promise for treating a range of human genetic diseases. While emerging clustered regularly interspaced short-palindromic repeats (CRISPR) technologies allow editing of the nuclear genome, it is still not possible to precisely manipulate mitochondrial DNA (mtDNA). Here, we summarize past developments and recent advances in nuclear and mitochondrial genome editing.

Keywords: gene therapy; genome editing; mitochondria; translational medicine.

Publication types

  • Research Support, N.I.H., Intramural
  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Gene Editing* / methods
  • Gene Editing* / trends
  • Genetic Therapy / methods
  • Genetic Therapy / trends
  • Genome
  • Genome, Mitochondrial
  • Humans
  • Translational Research, Biomedical