Evidence map›Paper›PMID 39569490›Full record

ArticleJournal of inherited metabolic disease2025

Endocannabinoid receptor 2 is a potential biomarker and therapeutic target for the lysosomal storage disorders.

Calogera M Simonaro, Makiko Yasuda, Edward H Schuchman

Abstract read
In one paragraph

Article in Journal of inherited metabolic disease, 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. Article
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

3 authors.

Calogera M SimonaroDepartment of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA.ORCID 0000-0003-3160-423X
Makiko YasudaDepartment of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
Edward H SchuchmanDepartment of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA.

Funding

Genetic Disease Foundation 0285-2690National MPS Society IF2669039Wylder Nation Foundation 0285-8019
6 · The paper itself

Abstract

Herein, we studied the expression of endocannabinoid receptor 2 (CB2R), a known inflammation mediator, in several lysosomal storage disorder (LSD) animal models and evaluated it as a potential biomarker and therapeutic target for these diseases. CB2R was highly elevated in the plasma of Farber disease and mucopolysaccharidosis (MPS) type IIIA mice, followed by Fabry disease and MPS type I mice. Mice with acid sphingomyelinase-deficient Niemann-Pick disease (ASMD) and rats with MPS type VI exhibited little or no plasma CB2R elevation. High-level expression of CB2R was also observed in tissues of Farber and MPS IIIA mice. Treatment of MPS IIIIA patient cells with CB2R agonists led to a reduction of CB2R and monocyte chemoattractant protein-1 (MCP-1), a chemotactic factor that is elevated in this LSD. Treatment of MPS IIIA mice with one of these agonists (JWH133) led to a reduction of plasma and tissue CB2R and MCP-1, a reduction of glial fibrillary acidic protein (GFAP) in the brain, and an improvement in hanging test performance. JWH133 treatment of Farber disease mice also led to a reduction of MCP-1 in tissues and plasma, and treatment of these mice by enzyme replacement therapy (ERT) led to a reduction of plasma CB2R, indicating its potential to monitor treatment response. Overall, these findings suggest that CB2R should be further examined as a potential therapeutic target for the LSDs and may also be a useful biomarker to monitor the impact of therapies.

Indexed as

BiomarkersDisease Models, AnimalLysosomal Storage DiseasesReceptor, Cannabinoid, CB2AnimalsCannabinoidsChemokine CCL2HumansMaleMiceMice, Inbred C57BLRats1,1-dimethylbutyl-1-deoxy-Delta(9)-THCBiomarkersCannabinoidsChemokine CCL2Receptor, Cannabinoid, CB2biomarkersendocannabinoidslysosomal storage disorderstherapeutic target

Identifiers

PMID39569490
PMCPMC11670223

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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.