| 发明名称 | Multiple exon skipping compositions for DMD | ||
| 摘要 | Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy. | ||
| 申请公布号 | US8865883(B2) | 申请公布日期 | 2014.10.21 |
| 申请号 | US201313830253 | 申请日期 | 2013.03.14 |
| 申请人 | Sarepta Therapeutics, Inc. | 发明人 | Sazani Peter;Kole Ryszard |
| 分类号 | C12N15/113;C12N15/11 | 主分类号 | C12N15/113 |
| 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 isolated antisense oligonucleotide of 20 to 35 nucleotides in length comprising at least 17 contiguous nucleotides of a nucleotide sequence set forth as SEQ ID NO: 2 wherein the oligonucleotide is capable of binding to human dystrophin pre-mRNA to induce exon 44 skipping, and wherein the oligonucleotide comprises a modification to resist degradation of an oligonucleotide:RNA heteroduplex by RNase H, and thymine bases (T) are optionally uracil bases (U). | ||
| 地址 | Bothell WA US | ||