Molecular therapy : the journal of the American Society of Gene TherapySep 12, 2021

Gene editing in living body cells using base and prime techniques

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 31, 2026

Abstract

Base editors and prime editors can effectively deliver precise DNA changes in animal models.

Base editors can correct the four most common single-base substitutions without causing extensive unwanted changes.
Prime editors can install any substitution, insertion, or deletion over a range of dozens of base pairs.
These editing technologies show greater efficiency and fewer byproducts compared to traditional methods that create double-strand DNA breaks.
Both viral and non-viral methods have been used to successfully deliver these genome editing tools in vivo.
The review highlights examples of somatic cell editing and discusses future development prospects.

Simplified

Recent advances in genome editing technologies have magnified the prospect of single-dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is anticipated to best treat patients. To install desired DNA changes with high precision, our laboratory developed base editors (BEs), which can correct the four most common single-base substitutions, and prime editors, which can install any substitution, insertion, and/or deletion over a stretch of dozens of base pairs. Compared to nuclease-dependent editing approaches that involve double-strand DNA breaks (DSBs) and often result in a large percentage of uncontrolled editing outcomes, such as mixtures of insertions and deletions (indels), larger deletions, and chromosomal rearrangements, base editors and prime editors often offer greater efficiency with fewer byproducts in slowly dividing or non-dividing cells, such as those that make up most of the cells in adult animals. Both viral and non-viral in vivo delivery methods have now been used to deliver base editors and prime editors in animal models, establishing that base editors and prime editors can serve as effective agents for in vivo therapeutic genome editing in animals. This review summarizes examples of in vivo somatic cell (post-natal) base editing and prime editing and prospects for future development.

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Competing interests

Declaration of interests The authors have filed patent applications on genome editing technologies through the Broad Institute of MIT and Harvard. D.R.L. is a consultant and cofounder of Beam Therapeutics, Prime Medicine, Pairwise Plants, Editas Medicine, and Chroma Medicine, companies that use genome editing or genome engineering, including base editing, prime editing, and epigenetic modification.

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Gene editing in living body cells using base and prime techniques

Abstract

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Competing interests

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You found one interesting study. We’ll send the next 7.

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