Evidence map›Paper›PMID 40625571›Full record

ReviewPediatric discovery2023

Gene therapy for pediatric genetic kidney diseases.

Yi Lu, Yandong Song, Shaokai Sun, Lirong Zhang, Yupeng Chen

Abstract readReview
In one paragraph

Review in Pediatric discovery, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
–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

5 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Review
  4. Assessing the potential ofPediatric discovery · 2024
    Article
  5. 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

5 authors.

Yi LuKey Laboratory of Immune Microenvironment and Disease (Ministry of Education) The Province and Ministry Co-sponsored Collaborative Innovation Center for Medical Epigenetics Department of Biochemistry and Molecular Biology School of Basic Medical Sciences Tianjin Institute of Urology The Second Hospital of Tianjin Medical University Tianjin Medical University Tianjin China.ORCID https://orcid.org/0000-0003-3469-2327
Yandong SongKey Laboratory of Immune Microenvironment and Disease (Ministry of Education) The Province and Ministry Co-sponsored Collaborative Innovation Center for Medical Epigenetics Department of Biochemistry and Molecular Biology School of Basic Medical Sciences Tianjin Institute of Urology The Second Hospital of Tianjin Medical University Tianjin Medical University Tianjin China.
Shaokai SunSchool of Medical Imaging Tianjin Medical University Tianjin China.
Lirong ZhangKey Laboratory of Immune Microenvironment and Disease (Ministry of Education) The Province and Ministry Co-sponsored Collaborative Innovation Center for Medical Epigenetics Department of Biochemistry and Molecular Biology School of Basic Medical Sciences Tianjin Institute of Urology The Second Hospital of Tianjin Medical University Tianjin Medical University Tianjin China.ORCID https://orcid.org/0000-0001-7466-1029
Yupeng ChenKey Laboratory of Immune Microenvironment and Disease (Ministry of Education) The Province and Ministry Co-sponsored Collaborative Innovation Center for Medical Epigenetics Department of Biochemistry and Molecular Biology School of Basic Medical Sciences Tianjin Institute of Urology The Second Hospital of Tianjin Medical University Tianjin Medical University Tianjin China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Genetic kidney disease is the main cause of chronic kidney disease in children. While the pathogenic genes associated with most genetic kidney diseases have been identified, the underlying mechanisms of disease initiation remain ambiguous, and effective treatment modalities are limited. Gene therapy has emerged as a promising approach for treating genetic diseases. Several gene therapy drugs have been successfully launched to treat genetic diseases affecting the eyes and muscles. However, the development of gene therapy agents for kidney diseases lags behind that of other genetic disorders. In this review, we first comprehensively summarize the main gene therapy strategies. Next, we provide an overview of current treatment options as well as the latest research on gene therapy approaches for pediatric genetic kidney diseases with a particular emphasis on Alport syndrome and polycystic kidney disease. Finally, we discuss the challenges and potential solutions for gene therapy of pediatric genetic kidney diseases.

Indexed as

AAVAlport syndromeCRISPR‐Cas9gene therapyLNPpediatric genetic kidney diseasesPKD

Identifiers

PMID40625571
PMCPMC12118231

What OpenQuestion holds

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Registered trials

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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.