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Home » Study casts dementia subtype as lysosomal storage disorder
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Study casts dementia subtype as lysosomal storage disorder

Aug. 26, 2021
By Anette Breindl
Researchers from Denali Therapeutics Inc. have identified new functional links between progranulin, lysosomal function, and a subtype of frontotemporal dementia caused by progranulin deficiency (FTD-GRN) that suggest progranulin-mediated FTD could be conceptualized as a lysosomal storage disorder (LSD). They also showed that delivery of their experimental therapeutic PTV:PGRN, also known as DNL-593, reduced cell damage and symptoms of FTD in cell and animal models.
BioWorld Science Neurology/psychiatric

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