ppanPersonalized Psychiatry and NeurologyPersonalized Psychiatry and Neurology2712-9179V. M. Bekhterev National Medical Research Centre for Psychiatry and Neurology of the Ministry of Health of the Russian Federation (Bekhterev NMRC PN)10.52667/2712-9179-2025-5-4-69-76ppan-148Research ArticleARTICLEEffectiveness and Safety of Intravenous Administration of Mesenchymal Stem Cells Overexpressing Arylsulfatase A for the Treatment of Metachromatic Leukodystrophy in a Pig ModelAyupovaAisilu I.

Kazan, 420008

FattakhovaAlisa A.

Kazan, 420008

SolovyevaValeria V.

Kazan, 420008

MukhamedshinaYana O.

Kazan, 420008; Kazan, 420111; 420012 Kazan

RizvanovAlbert A.

Kazan, 420008; Kazan, 420111

albert.rizvanov@kpfu.ruInstitute of Fundamental Medicine and Biology, Kazan Federal UniversityRussian FederationInstitute 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 UniversityRussian FederationInstitute of Fundamental Medicine and Biology, Kazan Federal University; Division of Medical and Biological Sciences, Tatarstan Academy of SciencesRussian Federation202524122025546976Copyright © Ayupova A.I., Fattakhova A.A., Solovyeva V.V., Mukhamedshina Y.O., Rizvanov A.A., 20252025Ayupova A.I., Fattakhova A.A., Solovyeva V.V., Mukhamedshina Y.O., Rizvanov A.A.Ayupova A.I., Fattakhova A.A., Solovyeva V.V., Mukhamedshina Y.O., Rizvanov A.A.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.jppn.ru/jour/article/view/148

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).

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The authors declare that there are no conflicts of interest present.