| 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 | US9035040(B2) | 申请公布日期 | 2015.05.19 |
| 申请号 | US201414317952 | 申请日期 | 2014.06.27 |
| 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.;Wallace Erika L. |
| 主权项 | 1. An antisense oligonucleotide of 25 nucleotides comprising at least 20 consecutive bases of C AUU CAA CUG UUG CCU CCG GUU CUG AAG GUG (SEQ ID NO: 193), in which cytosine bases are 5-methylcytosine bases, wherein the antisense oligonucleotide is a 2′-O-methyl phosphorothioate oligoribonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin pre-mRNA to induce exon 53 skipping. | ||
| 地址 | Crawley AU | ||