C9orf72 重复扩增导致的 ALS/FTD 的双向转录抑制治疗。
Bidirectional Transcriptional Inhibition as Therapy for ALS/FTD Caused by Repeat Expansion in C9orf72.
机构信息
Ludwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California at San Diego, La Jolla, CA 92093, USA.
Ludwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California at San Diego, La Jolla, CA 92093, USA; Department of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, CA 92093, USA.
出版信息
Neuron. 2016 Dec 21;92(6):1160-1163. doi: 10.1016/j.neuron.2016.12.008.
Hexanucleotide repeat expansion in the bi-directionally transcribed C9orf72 gene is the most frequent cause of familial ALS and frontotemporal dementia (FTD). Kramer et al. (2016) report in Science that targeted reduction in the transcription elongation factor SUPT4H1/SUPT5H reduces both sense and antisense repeat-containing RNAs and their associated neurodegeneration.
六核苷酸重复扩展在双向转录的 C9orf72 基因中是家族性肌萎缩侧索硬化症和额颞叶痴呆（FTD）最常见的原因。Kramer 等人（2016 年）在《科学》杂志上报道，靶向减少转录延伸因子 SUPT4H1/SUPT5H 可降低有义和反义重复 RNA 及其相关神经退行性变。
Zotero 插件