. Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis. J Neurosci. 2018 Feb 28;38(9):2341-2358. Epub 2018 Jan 29 PubMed.


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  1. Lysosomal Diseases: Stepping Stones to Gene Therapy for Alzheimer’s?