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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| Principais autores: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Elsevier
2025-09-01
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| coleção: | Molecular Therapy: Methods & Clinical Development |
| Assuntos: | |
| Acesso em linha: | http://www.sciencedirect.com/science/article/pii/S2329050125001226 |
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