Eck Randall J, Valdmanis Paul N, Liachko Nicole F, Kraemer Brian C
Graduate Program in Neuroscience, University of Washington, Seattle, WA, 98195, USA.
Division of Gerontology and Geriatric Medicine, Department of Medicine, University of Washington, Seattle, WA, 98104, USA.
Mol Brain. 2025 Jan 14;18(1):1. doi: 10.1186/s13041-025-01174-1.
Recent research has highlighted widespread dysregulation of alternative polyadenylation in amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration with TDP-43 pathology (FTLD-TDP). Here, we identify significant disruptions to 3` UTR polyadenylation in the ALS/FTLD-TDP mouse model rNLS8 that correlate with changes in gene expression and protein levels through the re-analysis of published RNA sequencing and proteomic data. A subset of these changes are shared with TDP-43 knock-down mice suggesting depletion of endogenous mouse TDP-43 is a contributor to polyadenylation dysfunction in rNLS8 mice. Some conservation exists between alternative polyadenylation in rNLS8 mice and human disease models including in disease relevant genes and biological pathways. Together, these findings support both TDP-43 loss and toxic gain-of-function phenotypes as contributors to the neurodegeneration in rNLS8 mice, nominating its continued utility as a preclinical model for investigating mechanisms of neurodegeneration in ALS/FTLD-TDP.
最近的研究突出了肌萎缩侧索硬化症(ALS)和具有TDP - 43病理特征的额颞叶变性(FTLD - TDP)中广泛存在的可变多聚腺苷酸化失调。在此,我们通过重新分析已发表的RNA测序和蛋白质组学数据,确定了ALS/FTLD - TDP小鼠模型rNLS8中3`UTR多聚腺苷酸化的显著破坏,这些破坏与基因表达和蛋白质水平的变化相关。这些变化中的一部分与TDP - 43敲低小鼠相同,表明内源性小鼠TDP - 43的耗竭是rNLS8小鼠多聚腺苷酸化功能障碍的一个原因。rNLS8小鼠的可变多聚腺苷酸化与人类疾病模型之间存在一些保守性,包括在疾病相关基因和生物学途径方面。总之,这些发现支持TDP - 43缺失和功能获得性毒性表型都是rNLS8小鼠神经退行性变的原因,表明其作为研究ALS/FTLD - TDP神经退行性变机制的临床前模型仍具有实用性。