Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis

Loss-of-function mutations in progranulin, a lysosomal glycoprotein, cause neurodegenerative disease. Progranulin haploinsufficiency causes frontotemporal dementia (FTD) and complete progranulin deficiency causes CLN11 neuronal ceroid lipofuscinosis (NCL). Progranulin replacement is a rational thera...

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發表在:J Neurosci
Main Authors: Arrant, Andrew E., Onyilo, Vincent C., Unger, Daniel E., Roberson, Erik D.
格式: Artigo
語言:Inglês
出版: Society for Neuroscience 2018
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Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5830520/
https://ncbi.nlm.nih.gov/pubmed/29378861
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.3081-17.2018
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