Stem cell and gene therapies for leukodystrophies
Leukodystrophies form a group of rare genetic disorders characterized by the progressive degeneration of white matter in the brain, often presenting in childhood with life-threatening consequences. Current therapeutic options are limited, particularly after symptom onset, and delayed diagnosis exace...
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| Format: | Article |
| Language: | English |
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Elsevier
2025-09-01
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| Series: | Molecular Therapy: Methods & Clinical Development |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050125001226 |
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| author | Wenhao Lin Min Zhang Sirui Zheng Qizhou Lian |
| author_facet | Wenhao Lin Min Zhang Sirui Zheng Qizhou Lian |
| author_sort | Wenhao Lin |
| collection | DOAJ |
| description | Leukodystrophies form a group of rare genetic disorders characterized by the progressive degeneration of white matter in the brain, often presenting in childhood with life-threatening consequences. Current therapeutic options are limited, particularly after symptom onset, and delayed diagnosis exacerbates disease progression. Stem cell and gene therapies have emerged as promising strategies to address these challenges. Neural stem cells (NSCs) and hematopoietic stem cells (HSCs) offer potential for CNS repair through differentiation into neurons and/or glial cells. Human pluripotent stem cells (hPSCs) provide a valuable model for studying disease mechanisms, drug screening, and therapeutic development. Additionally, mesenchymal stem cells (MSCs) exhibit immunomodulatory and secretory properties that may confer therapeutic benefits. Advanced gene therapies, including stem cell-based gene therapy and adeno-associated virus (AAV)-mediated gene replacement therapy (GRT), hold promise for curative interventions. This review highlights recent advances and challenges in stem cell and gene therapies for leukodystrophies, proposing a strategic integration of these approaches to maximize therapeutic efficacy by addressing their respective strengths and limitations. |
| format | Article |
| id | doaj-art-e129a2f95bf4446d890b1257dee3a0e1 |
| institution | Kabale University |
| issn | 2329-0501 |
| language | English |
| publishDate | 2025-09-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Therapy: Methods & Clinical Development |
| spelling | doaj-art-e129a2f95bf4446d890b1257dee3a0e12025-08-20T03:38:26ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012025-09-0133310152710.1016/j.omtm.2025.101527Stem cell and gene therapies for leukodystrophiesWenhao Lin0Min Zhang1Sirui Zheng2Qizhou Lian3Key Laboratory of Quantitative Synthetic Biology, Shenzhen Institute of Synthetic Biology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China; Faculty of Synthetic Biology, Shenzhen University of Advanced Technology, Shenzhen 518055, ChinaKey Laboratory of Quantitative Synthetic Biology, Shenzhen Institute of Synthetic Biology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China; Faculty of Synthetic Biology, Shenzhen University of Advanced Technology, Shenzhen 518055, ChinaKey Laboratory of Quantitative Synthetic Biology, Shenzhen Institute of Synthetic Biology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China; Faculty of Synthetic Biology, Shenzhen University of Advanced Technology, Shenzhen 518055, ChinaKey Laboratory of Quantitative Synthetic Biology, Shenzhen Institute of Synthetic Biology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China; Faculty of Synthetic Biology, Shenzhen University of Advanced Technology, Shenzhen 518055, China; Corresponding author: Qizhou Lian, Key Laboratory of Quantitative Synthetic Biology, Shenzhen Institute of Synthetic Biology, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China.Leukodystrophies form a group of rare genetic disorders characterized by the progressive degeneration of white matter in the brain, often presenting in childhood with life-threatening consequences. Current therapeutic options are limited, particularly after symptom onset, and delayed diagnosis exacerbates disease progression. Stem cell and gene therapies have emerged as promising strategies to address these challenges. Neural stem cells (NSCs) and hematopoietic stem cells (HSCs) offer potential for CNS repair through differentiation into neurons and/or glial cells. Human pluripotent stem cells (hPSCs) provide a valuable model for studying disease mechanisms, drug screening, and therapeutic development. Additionally, mesenchymal stem cells (MSCs) exhibit immunomodulatory and secretory properties that may confer therapeutic benefits. Advanced gene therapies, including stem cell-based gene therapy and adeno-associated virus (AAV)-mediated gene replacement therapy (GRT), hold promise for curative interventions. This review highlights recent advances and challenges in stem cell and gene therapies for leukodystrophies, proposing a strategic integration of these approaches to maximize therapeutic efficacy by addressing their respective strengths and limitations.http://www.sciencedirect.com/science/article/pii/S2329050125001226leukodystrophystem cell therapygene therapyhematopoietic stem cellneural stem cellmesenchymal stem cell |
| spellingShingle | Wenhao Lin Min Zhang Sirui Zheng Qizhou Lian Stem cell and gene therapies for leukodystrophies Molecular Therapy: Methods & Clinical Development leukodystrophy stem cell therapy gene therapy hematopoietic stem cell neural stem cell mesenchymal stem cell |
| title | Stem cell and gene therapies for leukodystrophies |
| title_full | Stem cell and gene therapies for leukodystrophies |
| title_fullStr | Stem cell and gene therapies for leukodystrophies |
| title_full_unstemmed | Stem cell and gene therapies for leukodystrophies |
| title_short | Stem cell and gene therapies for leukodystrophies |
| title_sort | stem cell and gene therapies for leukodystrophies |
| topic | leukodystrophy stem cell therapy gene therapy hematopoietic stem cell neural stem cell mesenchymal stem cell |
| url | http://www.sciencedirect.com/science/article/pii/S2329050125001226 |
| work_keys_str_mv | AT wenhaolin stemcellandgenetherapiesforleukodystrophies AT minzhang stemcellandgenetherapiesforleukodystrophies AT siruizheng stemcellandgenetherapiesforleukodystrophies AT qizhoulian stemcellandgenetherapiesforleukodystrophies |