ArticlePloS one2025
Volumetric MRI of dorsal root ganglia as a biomarker for disease progression and response to AAV treatment in a mouse model of Fabry disease.
Article in PloS one, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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.
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.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Noninvasive and objective biomarkers for disease-associated pathology are critical for clinical trials. For Fabry disease, one important pathological change due to the deficiency of the lysosomal enzyme α-galactosidase A (α-GAL) caused is accumulation of globotriaosylceramide (Gb3) in dorsal root ganglion (DRG) neurons, which manifests as the overall DRG hypertrophy. Magnetic resonance imaging (MRI) has been successfully used to noninvasively measure DRG enlargement in Fabry patients, and DRG volumetric MRI can be a potential noninvasive biomarker for Gb3 accumulations in DRG neurons in clinical trials. To evaluate disease progression and treatment response in preclinical proof-of-concept studies, we developed an in vivo MRI method to measure DRG size in the G3Stg/GLA knockout mouse model of Fabry disease. Compared to the wild type mice, the DRG enlargement in the Fabry mice was observed as early as 8 weeks of age, and a single administration of the human α-GAL-encoding adeno-associated virus (AAVGLA) normalized the enlarged DRG to the age-matched wild type mice. The DRG normalization was observed within 4 weeks of gene therapy (12 weeks of age) and was sustained up to 24 weeks of age. Furthermore, behavioral testing and histological/immunohistochemistry analyses of the DRG tissues corroborated the MRI findings. Volumetric DRG MRI has the sensitivity to measure Gb3 pathology-induced DRG volume changes and treatment response in live mice and can be a translational imaging biomarker in clinical trials for Fabry disease.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.