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Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders

Lysosomal storage diseases (LSDs) are a group of approximately 50 genetic disorders caused by mutations in genes coding enzymes that are involved in cell degradation and transferring lipids and other macromolecules. Accumulation of lipids and other macromolecules in lysosomes leads to the destructio...

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Autores principales: Issa, Shaza S., Shaimardanova, Alisa A., Valiullin, Victor V., Rizvanov, Albert A., Solovyeva, Valeriya V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8924473/
https://www.ncbi.nlm.nih.gov/pubmed/35308211
http://dx.doi.org/10.3389/fphar.2022.859516
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author Issa, Shaza S.
Shaimardanova, Alisa A.
Valiullin, Victor V.
Rizvanov, Albert A.
Solovyeva, Valeriya V.
author_facet Issa, Shaza S.
Shaimardanova, Alisa A.
Valiullin, Victor V.
Rizvanov, Albert A.
Solovyeva, Valeriya V.
author_sort Issa, Shaza S.
collection PubMed
description Lysosomal storage diseases (LSDs) are a group of approximately 50 genetic disorders caused by mutations in genes coding enzymes that are involved in cell degradation and transferring lipids and other macromolecules. Accumulation of lipids and other macromolecules in lysosomes leads to the destruction of affected cells. Although the clinical manifestations of different LSDs vary greatly, more than half of LSDs have symptoms of central nervous system neurodegeneration, and within each disorder there is a considerable variation, ranging from severe, infantile-onset forms to attenuated adult-onset disease, sometimes with distinct clinical features. To date, treatment options for this group of diseases remain limited, which highlights the need for further development of innovative therapeutic approaches, that can not only improve the patients’ quality of life, but also provide full recovery for them. In many LSDs stem cell-based therapy showed promising results in preclinical researches. This review discusses using mesenchymal stem cells for different LSDs therapy and other neurodegenerative diseases and their possible limitations.
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spelling pubmed-89244732022-03-17 Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders Issa, Shaza S. Shaimardanova, Alisa A. Valiullin, Victor V. Rizvanov, Albert A. Solovyeva, Valeriya V. Front Pharmacol Pharmacology Lysosomal storage diseases (LSDs) are a group of approximately 50 genetic disorders caused by mutations in genes coding enzymes that are involved in cell degradation and transferring lipids and other macromolecules. Accumulation of lipids and other macromolecules in lysosomes leads to the destruction of affected cells. Although the clinical manifestations of different LSDs vary greatly, more than half of LSDs have symptoms of central nervous system neurodegeneration, and within each disorder there is a considerable variation, ranging from severe, infantile-onset forms to attenuated adult-onset disease, sometimes with distinct clinical features. To date, treatment options for this group of diseases remain limited, which highlights the need for further development of innovative therapeutic approaches, that can not only improve the patients’ quality of life, but also provide full recovery for them. In many LSDs stem cell-based therapy showed promising results in preclinical researches. This review discusses using mesenchymal stem cells for different LSDs therapy and other neurodegenerative diseases and their possible limitations. Frontiers Media S.A. 2022-03-02 /pmc/articles/PMC8924473/ /pubmed/35308211 http://dx.doi.org/10.3389/fphar.2022.859516 Text en Copyright © 2022 Issa, Shaimardanova, Valiullin, Rizvanov and Solovyeva. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Pharmacology
Issa, Shaza S.
Shaimardanova, Alisa A.
Valiullin, Victor V.
Rizvanov, Albert A.
Solovyeva, Valeriya V.
Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
title Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
title_full Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
title_fullStr Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
title_full_unstemmed Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
title_short Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders
title_sort mesenchymal stem cell-based therapy for lysosomal storage diseases and other neurodegenerative disorders
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8924473/
https://www.ncbi.nlm.nih.gov/pubmed/35308211
http://dx.doi.org/10.3389/fphar.2022.859516
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