ASO-mediated knock-down of GPNMB in mutant-GRN and in Grn-deficient peripheral myeloid cells disrupts lysosomal function and immune responses.
在突变型 GRN 和 Grn 缺陷型外周髓系细胞中,ASO 介导的 GPNMB 敲低会破坏溶酶体功能和免疫反应
期刊:Molecular Neurodegeneration
影响因子:17.5
doi:10.1186/s13024-025-00829-w
Wallings Rebecca L, Gillett Drew A, Staley Hannah A, Mahn Savanna, Mark Julian, Neighbarger Noelle, Kordasiewicz Holly, Hirst Warren D, Tansey Malú Gámez