Evidence map›Paper›PMID 42496571›Full record

ArticleBiotech (Basel (Switzerland))2026

AAV Vector Toolkit for the Delivery and Expression of the Artificial microRNA in the Murine Heart.

Ivan I Galkin, Viktoriia V Skopenkova, Maria Y Shubina, Anna V Polikarpova, Svetlana G Vassilieva, Irina M Savchenko, Olga S Lebedeva, Daria V Goliusova, Margarita Y Sharikova, Vladimir V Gureev and 4 more

Abstract read
In one paragraph

Article in Biotech (Basel (Switzerland)), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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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

14 authors.

Ivan I GalkinLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.ORCID 0000-0002-9025-3794
Viktoriia V SkopenkovaLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.ORCID 0000-0001-7267-7967
Maria Y ShubinaLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.
Anna V PolikarpovaLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.ORCID 0000-0001-9442-8371
Svetlana G VassilievaLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.ORCID 0000-0002-0799-9844
Irina M SavchenkoMarlin Biotech, Sochi 354340, Russia.
Olga S LebedevaLopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, Moscow 119435, Russia.
Daria V GoliusovaLopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, Moscow 119435, Russia.ORCID 0000-0003-2837-8868
Margarita Y SharikovaLopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, Moscow 119435, Russia.ORCID 0009-0003-8678-4677
Vladimir V GureevLaboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod 308015, Russia.ORCID 0000-0003-1433-1225
Tatiana N MalorodovaLaboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod 308015, Russia.
Alexey V DeikinLaboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod 308015, Russia.
Tatiana V EgorovaLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.ORCID 0000-0002-3346-3242
Maryana V BardinaLaboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia.ORCID 0000-0003-1976-4767

Funding

Russian Science Foundation 26-14-00417The Ministry of Education and Science of the Russian Federation FFEW-2022-0001The Ministry of Education and Science of the Russian Federation FFEW-2025-0002The Ministry of Education and Science of the Russian Federation FZWG-2024-0003
6 · The paper itself

Abstract

backgroundAdeno-associated virus (AAV) vector-mediated gene transfer is an emerging treatment strategy for severe cardiac disorders with genetic etiology. We refined the AAV toolkit to achieve efficient and selective expression of the therapeutic transgene in mouse hearts.

methodsUsing vectors with a reporter transgene, we evaluated AAV administration routes, AAV serotype tropism to the myocardium, and cardiospecific promoters.

resultsWe showed that systemic AAV administration provides potent delivery and uniform transduction of cardiac tissue, outperforming localized injection techniques. The MyoAAV 2A capsid variant enabled an improved heart-to-liver transduction ratio compared to the parental AAV9 serotype. Screening a panel of cardiac and pan-muscular promoters in vitro and in vivo verified the superiority of the cardiac troponin T (cTnT) promoter for robust heart-specific transgene expression. Finally, we demonstrated that the cumulative properties of systemic AAV delivery, the MyoAAV 2A serotype, and the cTnT promoter allowed for efficient cardiac synthesis of the therapeutic transgene-an artificial miRNA designed for the gene suppression strategy of FLNC-related cardiomyopathy.

conclusionsOur findings establish an effective AAV approach for transgene transfer into the mouse heart and promote the development of gene therapy for cardiac disorders.

Indexed as

adeno-associated virus vectorscardiac diseasescardiac promotersgene therapymyotropic serotypestransgene delivery

Identifiers

PMID42496571
PMCPMC13398323

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LicenceCC BY
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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.