Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A
Abstract Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by an arylsulfatase A (ARSA) deficiency and characterized by severe neurological symptoms resulting from demyelination within the central and peripheral nervous systems. We investigated the feasibility and efficacy of...
| المؤلفون الرئيسيون: | , , , , , |
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| التنسيق: | مقال |
| اللغة: | English |
| منشور في: |
Nature Portfolio
2021-10-01
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| سلاسل: | Scientific Reports |
| الوصول للمادة أونلاين: | https://doi.org/10.1038/s41598-021-99979-2 |