ASO-mediated knock-down of GPNMB in mutant-GRN and in Grn-deficient peripheral myeloid cells disrupts lysosomal function and immune responses

ASO-mediated knock-down of GPNMB in mutant-GRN and in Grn-deficient peripheral myeloid cells disrupts lysosomal function and immune responses

Abstract Background GPNMB has been discussed as a potential therapeutic target in GRN-mediated neurodegeneration, based on the observed reproducible upregulation in FTD-GRN cerebrospinal fluid (CSF) and post-mortem brain. However, the functional impacts of up-regulated GPNMB are currently unknown, a...

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Bibliographic Details
Main Authors: Rebecca L. Wallings, Drew A. Gillett, Hannah A. Staley, Savanna Mahn, Julian Mark, Noelle Neighbarger, Holly Kordasiewicz, Warren D. Hirst, Malú Gámez Tansey
Format: Article
Language:English
Published: BMC 2025-04-01
Series:Molecular Neurodegeneration
Subjects:
Antisense oligonucleotide
Progranulin
Frontotemporal dementia
Neurodegeneration
GPNMB
Immune response
Online Access:https://doi.org/10.1186/s13024-025-00829-w
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