发明名称 |
Splice-region antisense composition and method |
摘要 |
Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5′ end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein. |
申请公布号 |
US8895722(B2) |
申请公布日期 |
2014.11.25 |
申请号 |
US201313859518 |
申请日期 |
2013.04.09 |
申请人 |
Sarepta Therapeutics, Inc. |
发明人 |
Iversen Patrick L.;Hudziak Robert |
分类号 |
C07H21/04;C07H21/02;A61K48/00 |
主分类号 |
C07H21/04 |
代理机构 |
Seed IP Law Group PLLC |
代理人 |
Seed IP Law Group PLLC |
主权项 |
1. An antisense compound composed of a 2′-O-methyl modified backbone and a base sequence of 12 to 25 nucleotide bases which is complementary to a target region within one exon of multiple exons of a preprocessed mRNA encoding a human protein, wherein the 5′-end of the target region is 12 bases downstream of a normal splice acceptor site in said preprocessed mRNA. |
地址 |
Corvallis OR US |