申请人:The General Hospital Corporation
公开号:US10961532B2
公开(公告)日:2021-03-30
Methods for reactivating genes on the inactive X chromosome that include administering one or both of a DNA methyltransferase (DNMT) Inhibitor and/or a topoisomerase inhibitor, e.g., etoposide and/or 5-azacytidine (aza), optionally in combination with an inhibitor of XIST RNA and/or an Xist-interacting protein, e.g., a chromatin-modifying protein, e.g., a small molecule or an inhibitory nucleic acid (such as a small inhibitory RNA (siRNAs) or antisense oligonucleotide (ASO)) that targets XIST RNA and/or a gene encoding an Xist-interacting protein, e.g., a chromatin-modifying protein.
重新激活无活性 X 染色体上基因的方法,包括施用 DNA 甲基转移酶(DNMT)抑制剂和/或拓扑异构酶抑制剂(如依托泊苷和/或 5-氮杂胞苷(azacytidine,aza))中的一种或两种,可选择与 XIST RNA 抑制剂和/或 Xist 交互蛋白(如染色质修饰蛋白)结合使用、染色质修饰蛋白,如靶向 XIST RNA 和/或 Xist 交互作用蛋白(如染色质修饰蛋白)编码基因的小分子或抑制性核酸(如小抑制性 RNA (siRNAs) 或反义寡核苷酸 (ASO))。