Evidence map›Paper›PMID 41275431›Full record

ArticleClinical and translational medicine2025

LNP-mediated in vivo base editing corrects Agxt to cure primary hyperoxaluria type 1.

Dexin Zhang, Rui Zheng, Zhoutong Chen, Xi Chen, Lei Yang, Yanan Huo, Yining Zhao, Jiaxin Huang, Dan Zhang, Shuming Yin and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Clinical and translational medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

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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

1 citing paper in PubMed.

  1. Article
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.

Dexin ZhangDepartment of Urology, Children's Hospital of Fudan University, Shanghai, China.
Rui ZhengDepartment of Urology, Children's Hospital of Fudan University, Shanghai, China.
Zhoutong ChenDepartment of Urology, Children's Hospital of Fudan University, Shanghai, China.
Xi ChenShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.
Lei YangHangzhou Institute of Medicine, Chinese Academy of Sciences, Hangzhou, China.
Yanan HuoShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.
Yining ZhaoDepartment of Urology, Affiliated Yantai Yuhuangding Hospital of Qingdao University, Yantai, China.
Jiaxin HuangDepartment of Urology, Children's Hospital of Fudan University, Shanghai, China.
Dan ZhangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.
Shuming YinShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.
Dali LiShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.
Hongquan GengDepartment of Urology, Children's Hospital of Fudan University, Shanghai, China.

Funding

Innovation Program of Shanghai Municipal Education Commission 2025GDZKZD03Shanghai Municipal Health Commission 20254Y0057the Foundation of Children's Hospital of Fudan University EKSJD202416the Foundation of Children's Hospital of Fudan University EKYX202423the National Clinical Key Specialty Construction Project 10000015Z155080000004the National Key Research and Development Program of China 2023YFC3403400the National Key Research and Development Program of China 2024YFC3406700the National Natural Science Foundation of China 32025023the National Natural Science Foundation of China 82470794the National Natural Science Foundation of China 82500860
6 · The paper itself

Abstract

objectivePrimary hyperoxaluria type 1 (PH1) is a rare autosomal recessive disorder caused by AGXT mutations, leading to hepatic oxalate overproduction, nephrolithiasis, and progressive renal failure. This study aims to evaluate the therapeutic potential of base editors delivered via lipid nanoparticles (LNPs) for treating PH1.

methodsWe utilized LNPs to deliver the base editor variant spG-ABE8e into a PH1 rat model. A single-dose injection of LNP-ABE was administered to assess its efficacy in correcting the pathogenic Agxt point mutation.

resultsTreatment with LNP-ABE achieved highly efficient correction of the Agxt mutation, which resulted in the normalization of urinary oxalate excretion, prevention of calcium oxalate deposits, and reversal of renal injury-associated gene expression profiles in PH1 rats. Furthermore, this study identified the minimum Agxt correction efficiency required for urinary oxalate normalization.

conclusionOur findings demonstrate that LNP-mediated delivery of base editors can effectively correct AGXT pathogenic mutations and ameliorate disease phenotypes in PH1, providing critical preclinical benchmarks for future clinical translation. KEY POINTS: The base editor precisely corrected the Agxt gene with high efficiency in PH1 rats. LNP-delivered Adenine Base Editor (ABE) normalized urinary oxalate levels and prevented calculus formation. This study identified the minimal Agxt correction efficiency required for urinary oxalate normalization.

Indexed as

Gene EditingHyperoxaluria, PrimaryNanoparticlesTransaminasesAnimalsDisease Models, AnimalMaleRatsRats, Sprague-DawleyAlanine-glyoxylate transaminaseTransaminasesalanine‐glyoxylate aminotransferase genebase editinggene editinglipid nanoparticleprimary hyperoxaluria type 1

Identifiers

PMID41275431
PMCPMC12640612

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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.