阅读说明：本技术 染色体9开放阅读框72基因表达的调节物及其用途 (Regulator for chromosome 9 open reading frame 72 gene expression and its use ) 是由 J.F.纳汉 A.帕塔马塔 M.萨米 于 2020-04-23 设计创作，主要内容包括：本公开提供用于调节有需要的患者中C9orf72基因转录的组合物及方法,所述患者包括患有C9orf72相关疾病诸如肌萎缩侧索硬化(ALS)和额颞叶痴呆(FTD)的患者。(The present disclosure provides compositions and methods for modulating C9orf72 gene transcription in patients in need thereof, including patients with C9orf72 associated diseases such as Amyotrophic Lateral Sclerosis (ALS) and frontotemporal dementia (FTD).)