Evidence map›Paper›PMID 40164763›Full record

ArticleNature biotechnology2026

Engineering a photoactivatable A-to-I RNA base editor for gene therapy in vivo.

Huiying Li, Yuhao Qiu, Bowen Song, Xinyi Quan, Dan Zhang, Xinru Li, Jingyun Yang, Xiaohong Liu, Zhiyang Zeng, Ji Jing and 11 more

Abstract read
PubMed Publisher
In one paragraph

Article in Nature biotechnology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing 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

9 citing papers in PubMed.

  1. Review
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  3. Traceless Regulation of Genetic Circuitry.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Review
  4. Article
  5. Review
  6. Article
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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

21 authors.

Huiying Li *Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Yuhao Qiu *Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Bowen Song *Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Xinyi Quan *Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Dan ZhangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Xinru LiShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Jingyun YangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Xiaohong LiuShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Zhiyang ZengSouthern Medical University Affiliated Fengxian Hospital, Shanghai, China.
Ji JingHangzhou Institute of Medicine (HIM), Chinese Academy of Sciences, Hangzhou, China.
Shuming YinShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Qi DaiDepartment of Molecular Bioscience, The Wenner-Gren Institute, Stockholm University, Stockholm, Sweden.ORCID http://orcid.org/0000-0002-2082-0693
Liren WangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.ORCID http://orcid.org/0000-0001-5264-0921
Honghui HanShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Haifeng YeShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.ORCID http://orcid.org/0000-0002-5482-8116
Zhenliang SunSouthern Medical University Affiliated Fengxian Hospital, Shanghai, China.
Yiyun ChengShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China.
Xueli ZhangSouthern Medical University Affiliated Fengxian Hospital, Shanghai, China.ORCID http://orcid.org/0000-0003-1715-7859
Bing DuShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China. bdu@bio.ecnu.edu.cn.ORCID http://orcid.org/0000-0002-5402-6527
Mingyao LiuShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China. myliu@bio.ecnu.edu.cn.ORCID http://orcid.org/0000-0001-7339-5048
Dali LiShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology and School of Life Sciences, East China Normal University, Shanghai Academy of Natural Sciences (SANS), Shanghai, China. dlli@bio.ecnu.edu.cn.ORCID http://orcid.org/0000-0002-0046-8493

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Tunable and reversible regulation of exogenous and endogenous gene expression would be useful for improving the safety and efficacy of gene therapy. Current chemically inducible systems are limited by the rapid diffusion and extended metabolism of small molecules, and associated side effects. Here we develop a photoactivatable RNA adenosine base editor (PA-rABE) by harnessing a compact Cas13 variant and a split ADAR2 deaminase fused with the Magnets system, which is activated through blue-light-induced dimerization. PA-rABE achieves highly efficient editing on endogenous RNA with minimal bystander editing and off-target effects. By editing a phosphorylation site of the endogenous CTNNB1 gene, PA-rABE stabilizes the β-catenin protein and activates Wnt signaling in vivo. Using adeno-associated virus vectors to deliver PA-rABE along with an hF9 variant containing a premature termination codon, we show amelioration of clotting defects in hemophilia B mice upon illumination. In summary, PA-rABE offers a controlled RNA base-editing technology for diverse biomedical applications, enabling reversible and spatiotemporally specific modulation.

Indexed as

AdenosineGene EditingGenetic TherapyRNA EditingAdenosine DeaminaseAnimalsbeta CateninDependovirusHEK293 CellsHumansMiceAdenosineAdenosine Deaminasebeta Catenin

Identifiers

What Socratic 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 Socratic graph.