Evidence map›Paper›PMID 39600089›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Amelioration of metabolic and behavioral defects through base editing in the Pah

Shuming Yin, Liangcai Gao, Xiaoyue Sun, Mei Zhang, Hongyi Gao, Xiaoqing Chen, Dan Zhang, Xinyu Ming, Lei Yang, Yaqiang Hu and 8 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Molecular therapy. Nucleic acids · 2025
    Review
  5. CRISPR-based functional genomics tools in vertebrate models.Experimental & molecular medicine · 2025
    Review
  6. 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

18 authors.

Shuming YinGuangdong Cardiovascular Institute, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, Southern Medical University, Guangzhou, Guangdong 510100, China; Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Liangcai GaoSchool of Life Sciences, East China Normal University, Shanghai 200241, China.
Xiaoyue SunShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Mei ZhangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Hongyi GaoShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Xiaoqing ChenShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Dan ZhangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Xinyu MingSchool of Life Sciences, East China Normal University, Shanghai 200241, China.
Lei YangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Yaqiang HuShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Xi ChenBRL Medicine, Inc., Shanghai 200241, China.
Meizhen LiuSchool of Life Sciences, East China Normal University, Shanghai 200241, China.
Xia ZhanDepartment of Pediatric Endocrinology and Genetics Metabolism, Shanghai Institute for Pediatric Research, Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China.
Yuting GuanShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Liren WangShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China.
Lianshu HanDepartment of Pediatric Endocrinology and Genetics Metabolism, Shanghai Institute for Pediatric Research, Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200092, China. Electronic address: hanlianshu@xinhuamed.com.cn.
Ping ZhuGuangdong Cardiovascular Institute, Guangdong Provincial People's Hospital, Guangdong Academy of Medical Sciences, Southern Medical University, Guangzhou, Guangdong 510100, China; Guangdong Provincial Key Laboratory of Pathogenesis, Targeted Prevention and Treatment of Heart Disease, Guangzhou Key Laboratory of Cardiac Pathogenesis and Prevention, Guangzhou, Guangdong 510080, China. Electronic address: tanganqier@163.com.
Dali LiShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China; School of Life Sciences, East China Normal University, Shanghai 200241, China. Electronic address: dlli@bio.ecnu.edu.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Phenylketonuria (PKU) is a liver metabolic disorder mainly caused by a deficiency of the hepatic phenylalanine hydroxylase (PAH) enzyme activity, often leading to severe brain function impairment in patients if untreated or if treatment is delayed. In this study, we utilized dual-AAV8 vectors to deliver a near PAM-less adenine base editor variant, known as ABE8e-SpRY, to treat the Pah

Indexed as

Disease Models, AnimalGene EditingPhenylalanine HydroxylasePhenylketonuriasAnimalsBehavior, AnimalBrainDependovirusGenetic TherapyGenetic VectorsHumansMiceMutationPhenylalaninePhenylalaninePhenylalanine HydroxylaseAAVABE8e-SpRYbase editinggene therapyliver metabolic disordersPahphenylketonuriaR408W mutationrain function defects

Identifiers

PMID39600089
PMCPMC11764323

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.