Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2019 Apr 10;27(4):735-746.
doi: 10.1016/j.ymthe.2019.01.014. Epub 2019 Jan 25.

Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9

Hong-Xia Zhang  1 , Ying Zhang  2 , Hao Yin  3
Affiliations
Review

Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9

Hong-Xia Zhang et al. Mol Ther. .

Abstract

Genome-editing technologies based on programmable nucleases have significantly broadened our ability to make precise and direct changes in the genomic DNA of various species, including human cells. Delivery of programmable nucleases into the target tissue or cell is one of the pressing challenges in transforming the technology into medicine. In vitro-transcribed (IVT) mRNA-mediated delivery of nucleases has several advantages, such as transient expression with efficient in vivo and in vitro delivery, no genomic integration, a potentially low off-target rate, and high editing efficiency. This review focuses on key barriers related to IVT mRNA delivery, on developed modes of delivery, and on the application and future prospects of mRNA encoding nuclease-mediated genome editing in research and clinical trials.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Genome Editing Mediated by Site-Specific Nucleases The zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), or CRISPR-Cas genome-editing nucleases are able to quickly search the genome, specifically bind various DNA sequences of interest, and efficiently induce double-strand breaks (DSBs). The DSBs are then effectively repaired by non-homologous end joining (NHEJ), or potentially homology-directed repair (HDR) if provided a DNA donor template, resulting in gene disruption, gene correction, or gene insertion, respectively.
Figure 2
Figure 2
Barriers for Non-viral Delivery Systemic delivery of genome-editing nucleases using non-viral vectors has to overcome various barriers.
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Capecchi M.R. Altering the genome by homologous recombination. Science. 1989;244:1288–1292. - PubMed
    1. Price B.D., D’Andrea A.D. Chromatin remodeling at DNA double-strand breaks. Cell. 2013;152:1344–1354. - PMC - PubMed
    1. Ceccaldi R., Rondinelli B., D’Andrea A.D. Repair Pathway Choices and Consequences at the Double-Strand Break. Trends Cell Biol. 2016;26:52–64. - PMC - PubMed
    1. Lieber M.R., Ma Y., Pannicke U., Schwarz K. Mechanism and regulation of human non-homologous DNA end-joining. Nat. Rev. Mol. Cell Biol. 2003;4:712–720. - PubMed
    1. Gaj T., Gersbach C.A., Barbas C.F., 3rd ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol. 2013;31:397–405. - PMC - PubMed

Publication types

LinkOut - more resources