Authors
Moksada Regmi, Kuiying Ma, Changhao Bi, Xueli Zhang, Dongdong Zhao, Lingling Yu, Huihui Yang, Ningli Wang, Chenlong Yang
Published in
Cell genomics. Volume 6. Issue 9. Pages 101298. Sep 09, 2026. Epub Jul 22, 2026.
Abstract
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.
PMID:
42486091
Bibliographic data and abstract were imported from PubMed on 09 Oct 2026.
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