Large-volume Intrathecal Enzyme Delivery Increases Survival of a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Late infantile neuronal ceroid lipofuscinosis (LINCL) is a progressive neurodegenerative lysosomal storage disorder caused by mutations in TPP1, the gene encoding the lysosomal protease tripeptidyl-peptidase (TPP1). LINCL primarily affects children, is fatal and there is no effective treatment. Admi...

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Main Authors: Xu, Su, Wang, Lingling, El-Banna, Mukarram, Sohar, Istvan, Sleat, David E, Lobel, Peter
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2011
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Original Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3188738/
https://ncbi.nlm.nih.gov/pubmed/21730969
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.130
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