Evidence map›Paper›PMID 40362629›Full record

ArticleInternational journal of molecular sciences2025

Allosteric Modulation of GCase Enhances Lysosomal Activity and Reduces ER Stress in GCase-Related Disorders.

Ilaria Fregno, Natalia Pérez-Carmona, Mikhail Rudinskiy, Tatiana Soldà, Timothy J Bergmann, Ana Ruano, Aida Delgado, Elena Cubero, Manolo Bellotto, Ana María García-Collazo and 1 more

Abstract read
In one paragraph

Article in International journal of molecular sciences, 2025. 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.

  1. Article
  2. Article
  3. Article
  4. Review
  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

11 authors.

Ilaria FregnoFaculty of Biomedical Sciences, Institute for Research in Biomedicine, Università Della Svizzera Italiana, CH-6500 Bellinzona, Switzerland.
Natalia Pérez-CarmonaGain Therapeutics, Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.
Mikhail RudinskiyFaculty of Biomedical Sciences, Institute for Research in Biomedicine, Università Della Svizzera Italiana, CH-6500 Bellinzona, Switzerland.ORCID 0000-0002-7915-5616
Tatiana SoldàFaculty of Biomedical Sciences, Institute for Research in Biomedicine, Università Della Svizzera Italiana, CH-6500 Bellinzona, Switzerland.
Timothy J BergmannFaculty of Biomedical Sciences, Institute for Research in Biomedicine, Università Della Svizzera Italiana, CH-6500 Bellinzona, Switzerland.
Ana RuanoGain Therapeutics, Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.
Aida DelgadoGain Therapeutics, Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0003-2793-1545
Elena CuberoGain Therapeutics, Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0002-2239-6670
Manolo BellottoGT Gain Therapeutics SA, CH-6900 Lugano, Switzerland.
Ana María García-CollazoGain Therapeutics, Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0009-0004-9582-7429
Maurizio MolinariFaculty of Biomedical Sciences, Institute for Research in Biomedicine, Università Della Svizzera Italiana, CH-6500 Bellinzona, Switzerland.ORCID 0000-0002-7636-5829

Funding

Eurostars-2 joint program with co-funding from the European Union Horizon 2020 Research and Innosuisse - Swiss Innovation Agency E!113321_CHAPERONESwiss National Science Foundation 310030-214903Swiss National Science Foundation 320030-227541
6 · The paper itself

Abstract

Variants in the

Indexed as

Endoplasmic Reticulum StressGaucher DiseaseGlucosylceramidaseLysosomesAllosteric RegulationFibroblastsHumansMutationParkinson DiseaseGBA protein, humanGlucosylceramidaseallosteric modulationGaucher diseaseglucosylceramidase beta 1 (GCase)lysosomal dysfunctionlysosomal storage disordersParkinson’s diseasepharmacological chaperonesprotein misfoldingsite-directed enzyme enhancement therapy (SEE-Tx)structurally targeted allosteric regulators (STARs)

Identifiers

PMID40362629
PMCPMC12072338

What OpenQuestion holds

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Registered trials

None linked

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.