Effectiveness and Safety of Intravenous Administration of Mesenchymal Stem Cells Overexpressing Arylsulfatase A for the Treatment of Metachromatic Leukodystrophy in a Pig Model
https://doi.org/10.52667/2712-9179-2025-5-4-69-76
Abstract
This study evaluated intravenous administration of allogeneic mesenchymal stem cells (MSCs) transduced with AAV9-ARSA to pigs. The MSCs were modified to overexpress human arylsulfatase A (ARSA). Thirty-five days after treatment, ARSA activity significantly increased in CNS tissues. No hepatotoxicity or systemic inflammation was observed. The results confirm the safety and efficacy of this MSC-based gene therapy for metachromatic leukodystrophy (MLD).
About the Authors
Aisilu A. Ayupova
Institute of Fundamental Medicine and Biology, Kazan Federal University
Russian Federation
Russian Federation
Kazan, 420008
Alisa A. Fattakhova
Institute of Fundamental Medicine and Biology, Kazan Federal University
Russian Federation
Russian Federation
Kazan, 420008
Valeria V. Solovyeva
Institute of Fundamental Medicine and Biology, Kazan Federal University
Russian Federation
Russian Federation
Kazan, 420008
Yana O. Mukhamedshina
Institute of Fundamental Medicine and Biology, Kazan Federal University; Division of Medical and Biological Sciences, Tatarstan Academy of Sciences; Department of Histology, Cytology, and Embryology, Kazan State Medical University
Russian Federation
Russian Federation
Kazan, 420008; Kazan, 420111; 420012 Kazan
Albert A. Rizvanov
Institute of Fundamental Medicine and Biology, Kazan Federal University; Division of Medical and Biological Sciences, Tatarstan Academy of Sciences
Russian Federation
Russian Federation
Kazan, 420008; Kazan, 420111
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Review
For citations:
Ayupova A.A., Fattakhova A.A., Solovyeva V.V., Mukhamedshina Ya.O., Rizvanov A.A. Effectiveness and Safety of Intravenous Administration of Mesenchymal Stem Cells Overexpressing Arylsulfatase A for the Treatment of Metachromatic Leukodystrophy in a Pig Model. Personalized Psychiatry and Neurology. 2025;5(4):69-76. https://doi.org/10.52667/2712-9179-2025-5-4-69-76
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