公开(公告)号：US20240093191A1

公开(公告)日：2024-03-21

申请号：US18274327

申请日：2022-02-03

申请人： Nizar Saad ,  Scott Quenton Harper

发明人： Nizar Saad ,  Scott Quenton Harper

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K45/06 ,  A61P21/00 ,  C12N15/86

CPC分类号： C12N15/113 ,  A61K9/0019 ,  A61K45/06 ,  A61P21/00 ,  C12N15/86 ,  C12N2310/141 ,  C12N2750/14143

摘要： Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression. Additionally, the disclosure provides an estrogen, synthetic estrogen, progesterone, progestin, melatonin, bleomycin, pyrazinamide, sorafenib, or a derivative thereof, or a combination of any thereof for upregulating expression of microRNA-675, inhibiting DUX4 expression, and for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.