Precision base-editing of the cryptic 3' acceptor site to correct the RNA splicing defect of β

Dan Lu, Xiuli Gong, Xinbing Guo, Qin Cai, Wenxiu Li, Yanwen Chen, Dali Li, Fanyi Zeng

Abstract read

In one paragraph

Article in Molecular biomedicine, 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

8 authors.

Dan LuShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China. danlu125@163.com.

Xiuli GongShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China.

Xinbing GuoShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China.

Qin CaiShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China.

Wenxiu LiShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China.

Yanwen ChenShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China.

Dali LiShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China. dlli@bio.ecnu.edu.cn.

Fanyi ZengShanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200040, China. fzeng@vip.163.com.

Funding

the Macau Science and Technology Development fund (FDCT) 003/2022/ALCthe Macau Science and Technology Development fund (FDCT) 0092/2022/A2the National Key Research and Development Program of China 2024YFC2707001the National Key Research and Development Program of China 2024YFC2707002the National Natural Science Foundation of China 31871484the National Natural Science Foundation of China 32570951the National Natural Science Foundation of China 82000186the National Natural Science Foundation of China 82271890the National Natural Science Foundation of China U23A20498the Natural Science Foundation of Shanghai 22ZR1451600

6 · The paper itself

Abstract

β-Thalassemia is one of the most common inherited disorders worldwide and is caused by mutations affecting β-globin production. β

Indexed as

beta-Globinsbeta-ThalassemiaGene EditingRNA Splice SitesRNA SplicingAnimalsCRISPR-Cas SystemsDisease Models, AnimalFemaleHumansMiceMutationbeta-GlobinsRNA Splice SitesBase editorGene therapyRNA splicingβ654-thalassemia

Identifiers

PMID42814316

DOI10.1186/s43556-026-00589-z

PMCPMC13627643

What OpenQuestion holds

Textmetadata

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.