ASO-mediated knockdown of GPNMB in mutant- GRN and Grn -deficient peripheral myeloid cells disrupts lysosomal function and immune responses.
Rebecca L WallingsDrew A GillettHannah A StaleySavanna MahnJulian MarkNoelle NeighbargerHolly KordasiewiczWarren D HirstMalú Gámez TanseyPublished in: bioRxiv : the preprint server for biology (2024)
would not be a rational therapeutic strategy because it is likely to dysregulate important immune cell effector functions.