Expanding the Toolbox and Targets for Gene Editing

Jiameng Dan; Sebastian Memczak; Juan Carlos Izpisua Belmonte

doi:10.1016/j.molmed.2020.12.005

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.

Authors

Jiameng Dan¹, Sebastian Memczak¹, Juan Carlos Izpisua Belmonte²

Affiliations

¹ Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.
² Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA. Electronic address: belmonte@salk.edu.

PMID: 33487569
DOI: 10.1016/j.molmed.2020.12.005

Abstract

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