Cell genomicsJul 22, 2026

Using base editing for precise medical treatments

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Jul 23, 2026

Abstract

Base editing (BE) may provide a method for correcting over half of known pathogenic genetic variants.

Base editing allows precise changes to DNA or RNA without causing double-strand breaks.
Recent improvements have enhanced the specificity, efficiency, and delivery of base editors for potential clinical applications.
Early successes of base editing have been noted in conditions such as sickle cell disease and beta-thalassemia.
Challenges remain, including unintended editing, delivery issues, and the need for long-term safety evidence.
Ongoing advancements and thorough evaluations are necessary to unlock the full potential of base editing in treating genetic diseases.

Simplified

Base editing (BE), the precise installation of single-nucleotide changes in DNA or RNA without inducing double-strand breaks, holds substantial therapeutic promise for correcting single-nucleotide variants, which constitute more than half of the known pathogenic genetic variants. Recent advances have improved base editor specificity, efficiency, and delivery, enabling clinically oriented procedures. Clinically, BE has shown early success or strong translational promise in sickle cell disease, β-thalassemia, leukemia (via CAR T and epitope engineering), hypercholesterolemia (PCSK9 and ANGPTL3), alpha-1-antitrypsin deficiency, and glycogen storage disease type Ia. Key remaining challenges include bystander editing within the activity window, residual off-target DNA and RNA editing, delivery constraints (payload size, tissue targeting, and redosing limits), immunogenicity, and the need for durable long-term safety evidence across relevant cell types and disease contexts. Continued technological refinements, careful preclinical validation, and rigorous clinical assessment will be essential to fully realize BE's transformative potential in precision medicine.

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Using base editing for precise medical treatments

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