申请人:GENEA BIOCELLS USA (HOLDINGS), INC.
公开号:US10933068B2
公开(公告)日:2021-03-02
Disclosed herein are methods and compositions for the treatment of facioscapulohumeral muscular dystrophy and other muscle diseases or disorders. In some cases, the methods and compositions involve the use of methyltransferase inhibitors to inhibit or repress DUX4 expression in muscle cells. Further disclosed herein are methods and cell based assays for screening compounds for the treatment of facioscapulohumeral muscular dystrophy and other muscle diseases.
本文公开了治疗面肱骨肌营养不良症和其他肌肉疾病或紊乱的方法和组合物。在某些情况下,这些方法和组合物涉及使用甲基转移酶抑制剂来抑制或抑制肌肉细胞中 DUX4 的表达。本文进一步公开了用于筛选治疗面阔肌营养不良症和其他肌肉疾病的化合物的方法和基于细胞的检测方法。