Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis

Classical late infantile neuronal ceroid lipofuscinosis (cLINCL) is a lysosomal storage disorder caused by mutations in CLN2, which encodes lysosomal tripeptidyl peptidase I (TPP1). Lack of TPP1 results in accumulation of autofluorescent storage material and curvilinear bodies in cells throughout th...

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izdano v:J Neurosci
Main Authors: Passini, Marco A., Dodge, James C., Bu, Jie, Yang, Wendy, Zhao, Qi, Sondhi, Dolan, Hackett, Neil R., Kaminsky, Stephen M., Mao, Qinwen, Shihabuddin, Lamya S., Cheng, Seng H., Sleat, David E., Stewart, Gregory R., Davidson, Beverly L., Lobel, Peter, Crystal, Ronald G.
Format: Artigo
Jezik:Inglês
Izdano: Society for Neuroscience 2006
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Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6675492/
https://ncbi.nlm.nih.gov/pubmed/16452657
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.2676-05.2006
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