Evidence map›Paper›PMID 40064881›Full record

ArticleNature communications2025

In vivo prime editing rescues photoreceptor degeneration in nonsense mutant retinitis pigmentosa.

Yidian Fu, Xiaoyu He, Liang Ma, Xin D Gao, Pengpeng Liu, Hanhan Shi, Peiwei Chai, Shengfang Ge, Renbing Jia, David R Liu and 2 more

Abstract read
In one paragraph

Article in Nature communications, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing 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

21 citing papers in PubMed.

  1. Prime Editing: An Overview.Methods in molecular biology (Clifton, N.J.) · 2027
    Review
  2. Role of Chromatin-Associated RNAs in Eye Physiology and Pathology.Methods in molecular biology (Clifton, N.J.) · 2027
    Review
  3. Review
  4. Review
  5. Article
  6. A primer on prime: A prime editing update from advances to first-in-human trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Review
  7. Article
  8. Article
  9. Gene-sized editing for the therapy of genetic diseases.Functional & integrative genomics · 2026
    Review
  10. Review
  11. Review
  12. Review
  13. Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. Review
  19. Gene Therapy Strategies for the Treatment of Bestrophinopathies.International journal of molecular sciences · 2025
    Review
  20. Review
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

12 authors.

Yidian Fu *Department of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.
Xiaoyu He *Department of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.
Liang Ma *Department of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.
Xin D GaoMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, 02142, USA.ORCID http://orcid.org/0000-0003-2917-2060
Pengpeng LiuInstitute of Advanced Biotechnology, Institute of Homeostatic Medicine, and School of Medicine, Southern University of Science and Technology, Shenzhen, 518055, China.
Hanhan ShiDepartment of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.
Peiwei ChaiDepartment of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.ORCID http://orcid.org/0000-0002-9135-0940
Shengfang GeDepartment of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.
Renbing JiaDepartment of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.ORCID http://orcid.org/0000-0001-6642-7451
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, 02142, USA.ORCID http://orcid.org/0000-0002-9943-7557
Xianqun FanDepartment of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China. fanxq@sjtu.edu.cn.ORCID http://orcid.org/0000-0002-9394-3969
Zhi YangDepartment of Ophthalmology, Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China. yangzhiscience@163.com.ORCID http://orcid.org/0000-0001-9440-7896

Funding

National Natural Science Foundation of China (National Science Foundation of China) 82200961
6 · The paper itself

Abstract

The next-generation gene editing tool, prime editing (PE), is adept at correcting point mutations precisely with high editing efficiency and rare off-target events and shows promising therapeutic value in treating hereditary diseases. Retinitis pigmentosa (RP) is the most common type of inherited retinal dystrophy and is characterized by progressive degeneration of retinal photoreceptors and, consequently, visual decline. To date, effective treatments for RP are lacking. Herein, a PE system is designed to target the PDE6B Y347X mutation in the rd1 mouse strain, a preclinical RP model. We screen and develop the PE system with epegRNA and RT

Indexed as

Codon, NonsenseCyclic Nucleotide Phosphodiesterases, Type 6Gene EditingRetinal DegenerationRetinitis PigmentosaAnimalsCRISPR-Cas SystemsDependovirusDisease Models, AnimalFemaleGenetic TherapyHumansMaleMiceMice, Inbred C57BLRetinal Rod Photoreceptor CellsCodon, NonsenseCyclic Nucleotide Phosphodiesterases, Type 6Pde6b protein, mouse

Identifiers

PMID40064881
PMCPMC11893901

What OpenQuestion holds

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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.