Evidence map›Paper›PMID 40725422›Full record

ArticleGenes2025

Comparative Evaluation of AAV8 and AAV9 Gene Therapy in Fabry Knockout (

Fu-Pang Chang, Ya-Ting Lee, Pao-Hsung Liu, Pei-Sin Chen, Yun-Ru Chen, Dau-Ming Niu

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Article in Genes, 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

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

1 citing paper in PubMed.

  1. 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

6 authors.

Fu-Pang ChangInstitute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan.
Ya-Ting LeeInstitute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan.
Pao-Hsung LiuInstitute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan.
Pei-Sin ChenInstitute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan.
Yun-Ru ChenDepartment of Pediatrics, Taipei Veterans General Hospital, Taipei 11217, Taiwan.ORCID 0000-0002-7410-0624
Dau-Ming NiuInstitute of Clinical Medicine, College of Medicine, National Yang Ming Chiao Tung University, Taipei 112304, Taiwan.ORCID 0000-0003-3962-1739

Funding

National Science and Technology Council 106-2320-B-075 -003 -MY3
6 · The paper itself

Abstract

backgroundFabry disease (FD) is an X-linked lysosomal storage disorder caused by mutations in the GLA gene, resulting in α-galactosidase A (α-Gal A) deficiency and progressive accumulation of globotriaosylceramide (Gb3). Current therapies, such as enzyme replacement and chaperone therapy, have limitations, including incomplete biodistribution and mutation-specific efficacy. Gene therapy using adeno-associated virus (AAV) vectors presents a promising alternative.

methodsIn this study, we assessed the dose-dependent effects of AAV8 and AAV9 vectors encoding human GLA in Gla knockout (

resultsAAV9-GLA produced significantly higher and more sustained enzyme activity than AAV8-GLA across plasma, liver, heart, and kidney. In symptomatic mice, AAV9-GLA achieved superior reductions in serum Gb3 and lyso-Gb3 levels, greater Gb3 clearance in heart and kidney tissues, and improved proteinuria. Anti-GLA IgG titers remained below threshold for the first four weeks and increased modestly by week eight, indicating a limited humoral immune response. No significant clinical signs or weight loss were observed in

conclusionsThese findings demonstrate that AAV9 provides enhanced biodistribution and therapeutic efficacy compared to AAV8, supporting its potential for the treatment of Fabry disease.

Indexed as

alpha-GalactosidaseDependovirusFabry DiseaseGenetic TherapyGenetic VectorsAnimalsDisease Models, AnimalEnzyme Replacement TherapyHumansMaleMiceMice, KnockoutTrihexosylceramidesalpha-GalactosidaseGLA protein, humanglobotriaosylceramideTrihexosylceramidesFabry diseasegene therapyGLA

Identifiers

PMID40725422
PMCPMC12294720

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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.