The present disclosure relates to compounds and methods for modulating the expression of bean (brain expressed, associated with NEDD4) and treating diseases and conditions in which bean plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence TGGAA; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence TGGAA; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.
本公开涉及化合物和方法,用于调节豆腐(与NED
D4相关的脑表达)的表达,并治疗豆腐发挥积极作用的疾病和病况。该化合物可以是一种转录调节分子,具有第一末端、第二末端和寡聚骨架,其中:a)第一末端包括一种DNA结合基团,能够非共价结合到核苷酸重复序列TGG
AA;b)第二末端包括结合到调节分子的蛋白结合基团,该调节分子调节包含核苷酸重复序列TGG
AA的
基因的表达;c)寡聚骨架包括连接第一末端和第二末端之间的连接物。