| 发明名称 | Modified polynucleotides for treating galactosylceramidase protein deficiency | ||
| 摘要 | The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules. | ||
| 申请公布号 | US9089604(B2) | 申请公布日期 | 2015.07.28 |
| 申请号 | US201414171249 | 申请日期 | 2014.02.03 |
| 申请人 | Moderna Therapeutics, Inc. | 发明人 | Chakraborty Tirtha;de Fougerolles Antonin;Weiss Ron |
| 分类号 | A61K48/00;C12N15/85;C07K14/47;C07K14/62;A61K31/7115;C12N9/88;A61K9/127;C12N15/11;C07K14/705;C07K14/505;C07K14/535;C12N9/64;C12N9/10;C12N9/00;C12N9/42;C12N15/87;C12P13/04;A61K38/45;C07K14/005;C12N9/16;C12N9/24;C12N15/52;A61K38/17;C07K14/435;C12P21/00;C07K14/485;A61K38/00 | 主分类号 | A61K48/00 |
| 代理机构 | DT Ward, PC | 代理人 | DT Ward, PC ;Ward Donna T.;Bryan Jennifer F. |
| 主权项 | 1. A method of producing a polypeptide of interest in a mammalian cell or tissue comprising contacting said mammalian cell or tissue with an mRNA having a 3′ untranslated region and a 5′ untranslated region, wherein said modified mRNA encodes a member selected from the group consisting of galactosylceramidase (SEQ ID NO: 47205), galactosylceramidase (SEQ ID NO: 47206), galactosylceramidase (SEQ ID NO: 47207), galactosylceramidase (SEQ ID NO: 47208) and galactosylceramidase (SEQ ID NO: 47209), and wherein said mRNA comprises a coding region, said coding region having at least 85% identity to SEQ ID NO: 82603. | ||
| 地址 | Cambridge MA US | ||