Evidence map›Paper›PMID 39445404›Full record

ArticleAnnals of medicine2024

Combined miRNA transcriptome and proteome analysis of extracellular vesicles in urine and blood from the Pompe mouse model.

David Merberg, Rodney Moreland, Zhenqiang Su, Bin Li, Bob Crooker, Kathleen Palmieri, Simon W Moore, Andrew Melber, Ruby Boyanapalli, Galen Carey and 1 more

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PubMed Full text at PMC Publisher
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Article in Annals of medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

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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

11 authors.

David MerbergTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.ORCID 0000-0002-5153-2889
Rodney MorelandTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.ORCID 0000-0003-0304-786X
Zhenqiang SuTakeda Pharmaceutical Company Limited, Preclinical and Translational Sciences, Cambridge, MA, USA.
Bin LiTakeda Pharmaceutical Company Limited, Preclinical and Translational Sciences, Cambridge, MA, USA.
Bob CrookerTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.
Kathleen PalmieriTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.
Simon W MooreTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.ORCID 0000-0003-1330-8381
Andrew MelberTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.
Ruby BoyanapalliTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.
Galen CareyTakeda Pharmaceutical Company Limited, Rare Disease Drug Discovery Unit, Cambridge, MA, USA.
Mahindra MakhijaTakeda International - UK, London, England.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionAcid α-glucosidase (GAA) is a lysosomal enzyme that hydrolyzes glycogen to glucose. Deficiency of GAA causes Pompe disease (PD), also known as glycogen storage disease type II. The resulting glycogen accumulation causes a spectrum of disease severity ranging from infantile-onset PD to adult-onset PD. Additional non-invasive biomarkers of disease severity are needed to monitor response to therapeutic interventions.

methodsWe measured protein and miRNA abundance in exosomes from serum and urine from the PD mouse model (B6;129-GaaTm1Rabn/J), wild-type mice, and PD mice treated with a candidate gene therapy.

resultsThere were significant differences in the abundance of 113 miRNA in serum exosomes from Pompe versus healthy mice. Levels of miR-206, miR-133, miR-1a, miR-486, and other important regulators of muscle development and maintenance were altered in the Pompe samples. The serum and urine exosome proteomes of healthy and Pompe mice also differed broadly. Several of the dysregulated proteins are encoded by genes with potential target sites for affected miRNA.

conclusionExosomes derived from urine or serum are a potential source of biomarkers for Pompe Disease. Further study of the differences in the miRNA transcriptome and proteome content of exosomes may yield new insights into disease mechanisms.

Indexed as

Disease Models, AnimalGlycogen Storage Disease Type IIMicroRNAsProteomeTranscriptomealpha-GlucosidasesAnimalsBiomarkersExosomesExtracellular VesiclesGenetic TherapyMaleMicealpha-GlucosidasesBiomarkersMicroRNAsProteomebiomarkerexosomesmiRNAPompe diseaseproteomes

Identifiers

PMID39445404
PMCPMC11504521

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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.