Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases

Ayupova, Aisylu I.; Sidorova, Angelina S.; Luzina, Ekaterina A.; Sufianov, Albert A.; Sufianova, Galina Z.; Zaynutdinov, Azat M.; Rizvanov, Albert A.; Solovyeva, Valeriya V.

doi:10.3390/cells15171540

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Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases

by

Aisylu I. Ayupova

¹

,

Angelina S. Sidorova

¹,

Ekaterina A. Luzina

¹,

Albert A. Sufianov

^2,3,

Galina Z. Sufianova

⁴,

Azat M. Zaynutdinov

⁵,

Albert A. Rizvanov

^1,6,*

and

Valeriya V. Solovyeva

^1,6

¹

Institute of Fundamental Medicine and Biology, Kazan Federal University, 420008 Kazan, Russia

²

Department of Neurosurgery, Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation (Sechenov University), 119991 Moscow, Russia

³

The Research and Educational Institute of Neurosurgery, Peoples’ Friendship University of Russia, 117198 Moscow, Russia

⁴

Department of Pharmacology, Tyumen State Medical University, 625023 Tyumen, Russia

⁵

Medical and Sanitary Unit of the Kazan Federal University, 420043 Kazan, Russia

⁶

Division of Medical and Biological Sciences, Academy of Sciences of the Republic of Tatarstan, 420111 Kazan, Russia

^*

Author to whom correspondence should be addressed.

Cells 2026, 15(17), 1540; https://doi.org/10.3390/cells15171540

Submission received: 24 July 2026 / Revised: 19 August 2026 / Accepted: 25 August 2026 / Published: 26 August 2026

(This article belongs to the Special Issue Mesenchymal Stem Cells in Cell Therapy, Regenerative Medicine, and Tissue Engineering)

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Abstract

Mesenchymal stromal cells (MSCs) have emerged as a promising therapeutic platform for central nervous system disorders, including neurodegenerative diseases and lysosomal storage disorders (LSDs). This review examines MSC mechanisms of action—paracrine activity, immunomodulation, antioxidant effects, TFEB-mediated autophagy regulation, and enzymatic cross-correction in LSDs—while critically assessing translational challenges. We provide a comparative analysis of MSC sources, administration routes, dosing regimens, and safety profiles, with emphasis on hemocompatibility and thrombotic risks. The evidence base for MSC efficacy in amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, and LSDs is systematically reviewed, highlighting both promising signals and limitations. MSC-derived extracellular vesicles are discussed as a cell-free alternative with improved safety and potential blood–brain barrier interaction. We propose an individualized monitoring framework integrating clinical scales, biomarkers, and neuroimaging. Despite preclinical promise, the field faces major hurdles: product standardization, optimal dosing, and the need for large, randomized controlled trials. The most rational path forward lies in combination strategies—MSCs as adjuncts to gene or enzyme replacement therapy—and engineered platforms for sustained delivery. This review provides a roadmap for translational decision-making and identifies critical gaps that must be addressed before MSC-based therapies can be integrated into routine neurological practice.

Keywords: mesenchymal stromal cells; extracellular vesicles; exosomes; neurodegenerative diseases; lysosomal storage disorders; cell therapy; immunomodulation; neuroinflammation; cross-correction; tissue factor; hemocompatibility

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MDPI and ACS Style

Ayupova, A.I.; Sidorova, A.S.; Luzina, E.A.; Sufianov, A.A.; Sufianova, G.Z.; Zaynutdinov, A.M.; Rizvanov, A.A.; Solovyeva, V.V. Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases. Cells 2026, 15, 1540. https://doi.org/10.3390/cells15171540

AMA Style

Ayupova AI, Sidorova AS, Luzina EA, Sufianov AA, Sufianova GZ, Zaynutdinov AM, Rizvanov AA, Solovyeva VV. Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases. Cells. 2026; 15(17):1540. https://doi.org/10.3390/cells15171540

Chicago/Turabian Style

Ayupova, Aisylu I., Angelina S. Sidorova, Ekaterina A. Luzina, Albert A. Sufianov, Galina Z. Sufianova, Azat M. Zaynutdinov, Albert A. Rizvanov, and Valeriya V. Solovyeva. 2026. "Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases" Cells 15, no. 17: 1540. https://doi.org/10.3390/cells15171540

APA Style

Ayupova, A. I., Sidorova, A. S., Luzina, E. A., Sufianov, A. A., Sufianova, G. Z., Zaynutdinov, A. M., Rizvanov, A. A., & Solovyeva, V. V. (2026). Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases. Cells, 15(17), 1540. https://doi.org/10.3390/cells15171540

Note that from the first issue of 2016, this journal uses article numbers instead of page numbers. See further details here.

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Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases

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