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Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic
Affiliation:1. Denali Therapeutics, South San Francisco, CA, USA;2. Memory and Aging Center, Department of Neurology, UCSF Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA;3. On behalf of the ALLFTD investigators;4. Department of Neurology, College of Medicine, Mayo Clinic, Rochester, MN, USA;5. Biochemistry and Molecular & Cellular Biology, Georgetown University, Washington, DC, USA
Abstract:
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  • Keywords:neurodegenerative disease  lysosome  lipids  lysobisphosphatidic acid  LBPA  galectin-3  GBA  lipofuscin  lipidomics  metabolomics
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