Evidence map›Paper›PMID 42486091›Full record

ReviewCell genomics2026

Base editing for precision therapeutics.

Moksada Regmi, Kuiying Ma, Changhao Bi, Xueli Zhang, Dongdong Zhao, Lingling Yu, Huihui Yang, Ningli Wang, Chenlong Yang

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Cell genomics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Moksada RegmiState Key Laboratory of Vascular Homeostasis and Remodeling, Department of Neurosurgery, Peking University Third Hospital, Peking University, Beijing, China; Center for Precision Neurosurgery and Oncology of Peking University Health Science Center, Peking University, Beijing, China; Center for Oculocranial Pressure Instability Disorders (COPID), Zhengzhou, Henan, China.
Kuiying MaChangping Laboratory, Beijing, China.
Changhao BiTianjin Institute of Industrial Biotechnology, Chinese Academy of Sciences, Tianjin, China.
Xueli ZhangTianjin Institute of Industrial Biotechnology, Chinese Academy of Sciences, Tianjin, China.
Dongdong ZhaoTianjin Institute of Industrial Biotechnology, Chinese Academy of Sciences, Tianjin, China.
Lingling YuChangping Laboratory, Beijing, China.
Huihui YangChangping Laboratory, Beijing, China.
Ningli WangCenter for Oculocranial Pressure Instability Disorders (COPID), Zhengzhou, Henan, China; Department of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing, China; Beijing Institute of Ophthalmology, Beijing Tongren Hospital, Beijing Key Laboratory of Intelligent Diagnosis Technology and Equipment for Optic Nerve-Related Eye Diseases, Capital Medical University, Beijing, China; Henan Academy of Innovations in Medical Science, Zhengzhou, Henan, China. Electronic address: wningli@vip.163.com.
Chenlong YangState Key Laboratory of Vascular Homeostasis and Remodeling, Department of Neurosurgery, Peking University Third Hospital, Peking University, Beijing, China; Center for Precision Neurosurgery and Oncology of Peking University Health Science Center, Peking University, Beijing, China; Center for Oculocranial Pressure Instability Disorders (COPID), Zhengzhou, Henan, China; Henan Academy of Innovations in Medical Science, Zhengzhou, Henan, China. Electronic address: vik.yang@pku.edu.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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.

Indexed as

Gene EditingGenetic TherapyPrecision MedicineAnimalsCRISPR-Cas SystemsHumansbase editingCRISPR-Cas systemsgene therapy deliverygenome engineeringprecision therapeuticssingle-nucleotide correction

Identifiers

PMID42486091
PMCPMC13576727

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.