| 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 | US9453225(B2) | 申请公布日期 | 2016.09.27 |
| 申请号 | US201514857590 | 申请日期 | 2015.09.17 |
| 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.;Harris Arianna D. |
| 主权项 | 1. An antisense oligonucleotide of formula (I): or a pharmaceutically acceptable salt thereof, wherein: Z is 18; R is H or —C(O)CH3, and each B is adenine, guanine, thymine, or cytosine, which taken together form a base sequence that is 100% complementary to 20 consecutive bases of exon 55 of the human dystrophin pre-mRNA, wherein the base sequence comprises 19 consecutive bases of TCTATGAGTTTCTTCCAAAGCAGCC (SEQ ID NO:527), and wherein the antisense oligonucleotide induces exon 55 skipping. | ||
| 地址 | Cambridge MA US | ||