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

Galkin, Ivan I.; Skopenkova, Viktoriia V.; Shubina, Maria Y.; Polikarpova, Anna V.; Vassilieva, Svetlana G.; Savchenko, Irina M.; Lebedeva, Olga S.; Goliusova, Daria V.; Sharikova, Margarita Y.; Gureev, Vladimir V.; Malorodova, Tatiana N.; Deikin, Alexey V.; Egorova, Tatiana V.; Bardina, Maryana V.

doi:10.3390/biotech15030055

Open AccessArticle

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

by

Ivan I. Galkin

^1,2,3

,

Viktoriia V. Skopenkova

^1,3

,

Maria Y. Shubina

^1,3,

Anna V. Polikarpova

^1,3

,

Svetlana G. Vassilieva

^1,3

,

Irina M. Savchenko

³,

Olga S. Lebedeva

⁴,

Daria V. Goliusova

⁴

,

Margarita Y. Sharikova

⁴

,

Vladimir V. Gureev

^2,†

,

Tatiana N. Malorodova

²,

Alexey V. Deikin

²,

Tatiana V. Egorova

^1,2,3

and

Maryana V. Bardina

^1,3,*

¹

Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology, Russian Academy of Sciences, Moscow 119334, Russia

²

Laboratory of Genetic Technologies and Genome Editing for Biomedicine and Animal Health, Joint Center for Genetic Technologies, Belgorod National Research University, Belgorod 308015, Russia

³

Marlin Biotech, Sochi 354340, Russia

⁴

Lopukhin Federal Research and Clinical Center of Physical-Chemical Medicine of Federal Medical Biological Agency, Moscow 119435, Russia

^*

Author to whom correspondence should be addressed.

^†

Current address: Federal State Budgetary Institution National Medical Research Center of Cardiology Named After Academician E.I. Chazov, Akademika Chazova St., 15A, Moscow 121552, Russia.

BioTech 2026, 15(3), 55; https://doi.org/10.3390/biotech15030055

Submission received: 18 May 2026 / Revised: 8 July 2026 / Accepted: 13 July 2026 / Published: 17 July 2026

(This article belongs to the Topic Advances in Gene Therapy of Human Diseases)

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Abstract

Background: Adeno-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. Methods: Using vectors with a reporter transgene, we evaluated AAV administration routes, AAV serotype tropism to the myocardium, and cardiospecific promoters. Results: We 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. Conclusions: Our findings establish an effective AAV approach for transgene transfer into the mouse heart and promote the development of gene therapy for cardiac disorders.

Keywords: adeno-associated virus vectors; cardiac diseases; transgene delivery; cardiac promoters; myotropic serotypes; gene therapy

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MDPI and ACS Style

Galkin, I.I.; Skopenkova, V.V.; Shubina, M.Y.; Polikarpova, A.V.; Vassilieva, S.G.; Savchenko, I.M.; Lebedeva, O.S.; Goliusova, D.V.; Sharikova, M.Y.; Gureev, V.V.; et al. AAV Vector Toolkit for the Delivery and Expression of the Artificial microRNA in the Murine Heart. BioTech 2026, 15, 55. https://doi.org/10.3390/biotech15030055

AMA Style

Galkin II, Skopenkova VV, Shubina MY, Polikarpova AV, Vassilieva SG, Savchenko IM, Lebedeva OS, Goliusova DV, Sharikova MY, Gureev VV, et al. AAV Vector Toolkit for the Delivery and Expression of the Artificial microRNA in the Murine Heart. BioTech. 2026; 15(3):55. https://doi.org/10.3390/biotech15030055

Chicago/Turabian Style

Galkin, Ivan I., 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 et al. 2026. "AAV Vector Toolkit for the Delivery and Expression of the Artificial microRNA in the Murine Heart" BioTech 15, no. 3: 55. https://doi.org/10.3390/biotech15030055

APA Style

Galkin, I. I., Skopenkova, V. V., Shubina, M. Y., Polikarpova, A. V., Vassilieva, S. G., Savchenko, I. M., Lebedeva, O. S., Goliusova, D. V., Sharikova, M. Y., Gureev, V. V., Malorodova, T. N., Deikin, A. V., Egorova, T. V., & Bardina, M. V. (2026). AAV Vector Toolkit for the Delivery and Expression of the Artificial microRNA in the Murine Heart. BioTech, 15(3), 55. https://doi.org/10.3390/biotech15030055

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

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