Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy
Metachromatic leukodystrophy (MLD) is a rare, autosomal recessive neurodegenerative disease caused by deficient activity of the lysosomal enzyme arylsulfatase A (ARSA), resulting in sulfatide accumulation and subsequent demyelination and neuronal damage within the central and peripheral nervous syst...
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| Autors principals: | , , , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Elsevier
2024-06-01
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| Col·lecció: | Molecular Therapy: Methods & Clinical Development |
| Matèries: | |
| Accés en línia: | http://www.sciencedirect.com/science/article/pii/S2329050124000640 |
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