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: Spampanato, Carmine, De Leonibus, Elvira, Dama, Paola, Gargiulo, Annagiusi, Fraldi, Alessandro, Sorrentino, Nicolina Cristina, Russo, Fabio, Nusco, Edoardo, Auricchio, Alberto, Surace, Enrico M, Ballabio, Andrea
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2011
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Original Article
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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