Evidence map›Paper›PMID 41821311›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026

Nanoparticles targeting liver sinusoidal endothelial cells improve tolerance to vector and transgene antigens through tolerance spreading.

Romain Hardet, Shu-Hung Wang, Sandrine Delignat, Marine Blandin, Reinaldo Digigow, Cornelia Gottwick, Markus Heine, Lígia Margarida Marques Mesquita, Marco Fanzutti, Anna-Lisa Vocaturo and 5 more

Abstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

15 authors.

Romain HardetUniversity Rouen Normandie, INSERM, Normandie University, PANTHER, UMR 1234, 76000 Rouen, France.
Shu-Hung WangTopas Therapeutics GmbH, Hamburg, Germany.
Sandrine DelignatInstitut National de la Santé et de la Recherche Médicale, Centre de Recherche des Cordeliers, CNRS, Sorbonne Université, Université de Paris, 75006 Paris, France.
Marine BlandinUniversity Rouen Normandie, INSERM, Normandie University, PANTHER, UMR 1234, 76000 Rouen, France.
Reinaldo DigigowTopas Therapeutics GmbH, Hamburg, Germany.
Cornelia GottwickDepartment of Medicine, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.
Markus HeineDepartment of Biochemistry and Molecular Cell Biology (N30), University Medical Clinic Hamburg-Eppendorf, Hamburg, Germany.
Lígia Margarida Marques MesquitaTopas Therapeutics GmbH, Hamburg, Germany.
Marco FanzuttiTopas Therapeutics GmbH, Hamburg, Germany.
Anna-Lisa VocaturoTopas Therapeutics GmbH, Hamburg, Germany.
Disha MungalparaTopas Therapeutics GmbH, Hamburg, Germany.
Olivier BoyerUniversity Rouen Normandie, INSERM, Normandie University, PANTHER, UMR 1234, 76000 Rouen, France; Department of Immunology and Biotherapy, CHU de Rouen, Rouen, France.
Sébastien Lacroix-DesmazesInstitut National de la Santé et de la Recherche Médicale, Centre de Recherche des Cordeliers, CNRS, Sorbonne Université, Université de Paris, 75006 Paris, France.
Sabine FleischerTopas Therapeutics GmbH, Hamburg, Germany. Electronic address: fleischer@topas-therapeutics.com.
Sahil AdriouchUniversity Rouen Normandie, INSERM, Normandie University, PANTHER, UMR 1234, 76000 Rouen, France. Electronic address: sahil.adriouch@univ-rouen.fr.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Liver sinusoidal endothelial cells (LSECs) naturally cross-present antigens and induce T cell tolerance. Targeting LSECs with peptide-coupled nanoparticles offers an efficient strategy to induce antigen-specific immune tolerance. Previous preclinical and clinical studies have shown that peptide-coupled nanoparticles can effectively inhibit T cell responses to the selected cognate peptide epitopes. However, clinical situations such as viral-vector-mediated gene therapy would benefit from simultaneous tolerance induction to multiple epitopes/antigens, posing a significant challenge. In this study, we used mouse models of adeno-associated virus (AAV)-mediated gene transfer to assess the in vivo effects of peptide-loaded nanoparticles designed to tolerize immune responses to transgene- or/and capsid-derived epitopes. We report here for the first time that LSEC-targeting nanoparticles coupled to a single peptide epitope promoted extension of tolerance to multiple relevant epitopes/antigens and simultaneously tolerized both CD4

Indexed as

AntigensEndothelial CellsGenetic VectorsImmune ToleranceLiverNanoparticlesTransgenesAnimalsCD4-Positive T-LymphocytesCD8-Positive T-LymphocytesDependovirusMicePeptidesAntigensPeptidesAAVadeno-associated viral vectorsimmune toleranceliver sinusoidal endothelial cellsLSECsnanoparticlestolerance spreadingtransgene persistence

Identifiers

PMID41821311
PMCPMC13330011

What OpenQuestion holds

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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.