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Efficacy of a Combined Intracerebral and Systemic Gene Delivery Approach for the Treatment of a Severe Lysosomal Storage Disorder
Multiple sulfatase deficiency (MSD), a severe autosomal recessive disease is caused by mutations in the sulfatase modifying factor 1 gene (Sumf1). We have previously shown that in the Sumf1 knockout mouse model (Sumf1(−/−)) sulfatase activities are completely absent and, similarly to MSD patients, t...
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| Main Authors: | , , , , , , , , , , |
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| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
Nature Publishing Group
2011
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3098632/ https://ncbi.nlm.nih.gov/pubmed/21326216 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.299 |
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