| 发明名称 | Antisense oligonucleotides for inducing exon skipping and methods of use thereof | ||
| 摘要 | An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202. | ||
| 申请公布号 | US9422555(B2) | 申请公布日期 | 2016.08.23 |
| 申请号 | US201514857555 | 申请日期 | 2015.09.17 |
| 申请人 | The University of Western Australia | 发明人 | Wilton Stephen Donald;Fletcher Sue;McClorey Graham |
| 分类号 | C07H21/04;C12N15/113 | 主分类号 | C07H21/04 |
| 代理机构 | Nelson Mullins Riley & Scarborough LLP | 代理人 | Nelson Mullins Riley & Scarborough LLP ;Mandragouras, Esq. Amy E.;Harris Ariana D. |
| 主权项 | 1. An antisense oligonucleotide of 25 bases comprising a base sequence that is 100% complementary to 25 consecutive bases of exon 45 of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 12 consecutive bases of CCAAUGCCAUCCUGGAGUUCCUGUAA (SEQ ID NO: 207), in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′-O-methyl phosphorothioate oligoribonucleotide, and wherein the antisense oligonucleotide induces exon 45 skipping; or a pharmaceutically acceptable salt thereof. | ||
| 地址 | Crawley AU | ||