| 发明名称 | Compound and method for treating myotonic dystrophy | ||
| 摘要 | Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1. | ||
| 申请公布号 | US8835402(B2) | 申请公布日期 | 2014.09.16 |
| 申请号 | US201314038314 | 申请日期 | 2013.09.26 |
| 申请人 | Sarepta Therapeutics, Inc. | 发明人 | Kole Ryszard;Hanson Gunnar J. |
| 分类号 | C12N15/113 | 主分类号 | C12N15/113 |
| 代理机构 | Seed IP Law Group PLLC | 代理人 | Seed IP Law Group PLLC |
| 主权项 | 1. An antisense compound for treating myotonic dystrophy DM1, consisting of a 9-base phosphorodiamidate morpholino antisense oligonucleotide, wherein at least one and up to about 1 per every 2 intersubunit linkage(s) contains a pendant cationic group, and wherein the 9 bases are complementary to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA and have the base sequence of SEQ ID NO:5 or SEQ ID NO:9. | ||
| 地址 | Corvallis OR US | ||