Evidence map›Paper›PMID 39743356›Full record

ReviewZhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology2025

[Research progresses in gene therapy for hepatolenticular degeneration].

G Chen, H Y Zheng, F Liu, J Yuan, Y H Xu, W S Cheng

Abstract readReviewEnglish Abstract
PubMed Full text at PMC Publisher
In one paragraph

Review in Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology, 2025. 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

6 authors.

G ChenPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China Department of Clinical Laboratory, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China Anhui Province Key Laboratory of Zoonoses (Anhui Medical University), Hefei 230032, China The Provincial Key Laboratory of Zoonoses of High Institutions in Anhui (Anhui Medical University), Hefei 230032, China.
H Y ZhengPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China Department of Clinical Laboratory, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China Anhui Province Key Laboratory of Zoonoses (Anhui Medical University), Hefei 230032, China The Provincial Key Laboratory of Zoonoses of High Institutions in Anhui (Anhui Medical University), Hefei 230032, China.
F LiuDepartment of Clinical Laboratory, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China Anhui Province Key Laboratory of Zoonoses (Anhui Medical University), Hefei 230032, China The Provincial Key Laboratory of Zoonoses of High Institutions in Anhui (Anhui Medical University), Hefei 230032, China.
J YuanPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China NHC Key Laboratory of Study on Abnormal Gametes and Reproductive Tract (Anhui Medical University), Hefei 230032, China Engineering Research Center of Biopreservation and Artificial Organs, Ministry of Education, Hefei 230032, China Key Laboratory of Population Health Across Life Cycle (Anhui Medical University), Ministry of Education of the People's Republic of China, Hefei 230032, China Anhui Province Key Laboratory of Reproductive Disorders and Obstetrics and Gynecology Diseases, Hefei 230032, China Biopreservation and Artificial Organs, Anhui Provincial Engineering Research Center, Anhui Medical University, Hefei 230032, China Anhui Provincial Institute of Translational Medicine, Hefei 230032, China.
Y H XuDepartment of Clinical Laboratory, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China Anhui Province Key Laboratory of Zoonoses (Anhui Medical University), Hefei 230032, China The Provincial Key Laboratory of Zoonoses of High Institutions in Anhui (Anhui Medical University), Hefei 230032, China.
W S ChengPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, Hefei 230022, China NHC Key Laboratory of Study on Abnormal Gametes and Reproductive Tract (Anhui Medical University), Hefei 230032, China Engineering Research Center of Biopreservation and Artificial Organs, Ministry of Education, Hefei 230032, China Key Laboratory of Population Health Across Life Cycle (Anhui Medical University), Ministry of Education of the People's Republic of China, Hefei 230032, China Anhui Province Key Laboratory of Reproductive Disorders and Obstetrics and Gynecology Diseases, Hefei 230032, China Biopreservation and Artificial Organs, Anhui Provincial Engineering Research Center, Anhui Medical University, Hefei 230032, China Anhui Provincial Institute of Translational Medicine, Hefei 230032, China.

Funding

Anhui Provincial Natural Science Foundation 2308085QH244China Postdoctoral Science Foundation 2023M740023Funded Project for Scientific Research Activities of Postdoctoral Researchers in Anhui Province 2022A574National Natural Science Foundation of China 82101954, 82302101Scientific Research Fund of Anhui Institute of Translational Medicine 2023zhyx-C23Scientific Research Project of Universities in Anhui Province 2022AH051161
6 · The paper itself

Abstract

Hepatolenticular degeneration, also known as Wilson's disease, is a type of autosomal recessive genetic disorder of copper metabolism. The causative gene, ATP7B, is located on the long arm of chromosome 13 and encodes a P-type ATPase that is involved in copper transport. Pathogenic mutations in the ATP7B gene sequence lead to the diminished or lost function of the ATP7B protein, resulting in pathological copper deposition in organs such as the liver, brain, kidneys, and cornea. Currently, the treatment of Wilson's disease primarily involves oral medications to promote copper excretion or reduce copper absorption so as to alleviate the state of illness. However, pharmacological treatment has objective limitations, including the need for lifelong therapy and varying degrees of adverse drug reactions in some patients. Gene therapy can fully correct the genetic defect, restore ATP7B protein function, achieve a curative effect, and improve the patient's quality of life.

Indexed as

Adenosine TriphosphatasesCation Transport ProteinsGenetic TherapyHepatolenticular DegenerationCopper-Transporting ATPasesHumansAdenosine TriphosphatasesATP7B protein, humanCation Transport ProteinsCopper-Transporting ATPases

Identifiers

PMID39743356
PMCPMC12899212

What OpenQuestion holds

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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.