ACGT letters graphic representing DNA code

Gene editing

Understand a gene-therapy approach that aims to modify DNA

Gene editing can potentially alter a patient’s DNA by targeting and modifying selected genes.1 While genetic engineering and DNA manipulation date back to the 1970s, the development of gene editing has accelerated over the past 10 years with ongoing research and new applications for several technologies.1-3 Stay informed as the gene-editing field continues to advance.

ACGT letters graphic representing DNA code

Gene editing

Understand a gene-therapy approach that aims to modify DNA

Gene editing can potentially alter a patient’s DNA by targeting and modifying selected genes.1 While genetic engineering and DNA manipulation date back to the 1970s, the development of gene editing has accelerated over the past 10 years with ongoing research and new applications for several technologies.1-3 Stay informed as the gene-editing field continues to advance.

New technologies, new possibilities

Technologies currently under research enable addition or deletion of genes, or changes to small sections of DNA.4 This can occur by introducing gene-editing tools in cells through nuclease-based techniques that slice DNA, spur a repair, and set a programmed sequence change in motion.5 These technologies may make it possible to rewrite snippets of DNA code and repair disease-causing gene mutations, alter small sections of a gene, or refine gene expression.6

Gene-editing technologies currently under clinical investigation

CRISPR/Cas system gene editing a DNA strand

CRISPR: Clustered regularly interspaced short palindromic repeats

These systems leverage a CRISPR-associated (Cas) nuclease enzyme plus a guide RNA to deliver targeted, precise, and site-specific gene editing.6,7

TALEN technology gene editing a DNA strand

TALENs: Transcription activator-like effector nucleases

Instead of an RNA guide, TALENs utilize proteins to target and then bind to specific segments of DNA. TALEN technology requires new protein design, production, and validation with each application.6,8

ZFN technology gene editing a DNA strand

ZFNs: Zinc-finger nucleases

Structurally similar to TALENs, ZFNs consist of a chain of zinc-finger proteins fused with a bacterial nuclease, generating a system that targets specific sites within the DNA sequence.6,9

Base editing system gene editing a DNA strand

Base editing

Base editing combines a CRISPR/Cas system with a base editor to precisely and irreversibly convert one base pair to another. This system works without the DNA double-strand break that CRISPR/Cas tools use, and thus does not rely on damage response mechanisms.1,7

Meganuclease technology gene editing a DNA strand

Other technologies

The field of gene editing has a number of other medical technologies in development, including homing endonucleases and meganucleases (MegNs) that target and cleave DNA sequences, and prime editing that generates RNA templates for gene alteration.6,10

Learn about CRISPR/Cas gene editing

Explore gene editing in more detail by reviewing one specific approach.

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References:  1. Li H, Yang Y, Hong W, Huang M, Wu M, Zhao X. Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects. Signal Transduct Target Ther. 2020;5(1):1. doi:10.1038/s41392-019-0089-y.  2. Gene therapy. ClinicalTrials.gov. Accessed April 14, 2023. https://clinicaltrials.gov/ct2/results?cond=&term=gene+therapy&cntry=US&state=&city=&dist.  3. Approved cellular and gene therapy products. US Food & Drug Administration. Updated October 26, 2021. Accessed April 14, 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.  4. Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age. Science. 2018;359(6372):eaan4672. doi:10.1126/science.aan4672.  5. Bulaklak K, Gersbach CA. The once and future gene therapy. Nat Commun. 2020;11(1):5820.  doi:10.1038/s41467-020-19505-2.  6. Khalil AM. The genome editing revolution: review. J Genet Eng Biotechnol. 2020;18(1):68. doi:10.1186/s43141-020-00078-y.  7. Uddin F, Rudin CM, Sen T. CRISPR gene therapy: applications, limitations, and implications for the future. Front Oncol. 2020;10:1387. doi:10.3389/fonc.2020.01387.  8. Barrangou R, Doudna JA. Applications of CRISPR technologies in research and beyond. Nat Biotechnol. 2016;34(9):933-941. doi:10.1038/nbt.3659.  9. Janik E, Niemcewicz M, Ceremuga M, Krzowski L, Saluk-Bijak J, Bijak M. Various aspects of a gene editing system—CRISPR–Cas9. Int J Mol Sci. 2020;21(24):9604. doi:10.3390/ijms21249604.  10. Doudna JA. The promise and challenge of therapeutic genome editing. Nature. 2020;578(7794):229-236. doi:10.1038/s41586-020-1978-5.