发明名称 EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
摘要 Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
申请公布号 US2014329881(A1) 申请公布日期 2014.11.06
申请号 US201414214480 申请日期 2014.03.14
申请人 Sarepta Therapeutics, Inc. 发明人 Bestwick Richard K.;Frank Diane Elizabeth;Schnell Fred Joseph
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项 1. An antisense oligomer of 20-50 nucleotides in length capable of binding a selected target to induce exon skipping in the human dystrophin gene, wherein said antisense oligomer comprises a sequence of bases that specifically hybridizes to an exon 44 target region selected from the group consisting of H44A(−07+15), H44A(−08+15), H44A(−06+15), H44A(−08+17), H44A(−07+17), and H44A(−06+17), wherein the bases of said oligomer are linked to morpholino ring structures, and wherein said morpholino ring structures are joined by phosphorous-containing intersubunit linkages joining a morpholino nitrogen of one ring structure to a 5′ exocyclic carbon of an adjacent ring structure.
地址 Bothell WA US