| 发明名称 |
MODIFIED POLYNUCLEOTIDES FOR TREATING DOLICHYL-PHOSPHATE (UDP-N-ACETYLGLUCOSAMINE) N-ACETYLGLUCOSAMINEPHOSPHOTRANSFERASE 1 (GLCNAC-1-P TRANSFERASE) 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 |
US2014179771(A1) |
申请公布日期 |
2014.06.26 |
| 申请号 |
US201414171119 |
申请日期 |
2014.02.03 |
| 申请人 |
Moderna Therapeutics, Inc. |
发明人 |
Bancel Stephane;Chakraborty Tirtha;de Fougerolles Antonin;Whoriskey Susan |
| 分类号 |
A61K38/45 |
主分类号 |
A61K38/45 |
| 代理机构 |
|
代理人 |
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| 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 a modified mRNA, wherein said modified mRNA encodes dolichyl-phosphate (UDP-N-acetylglucosamine) N-acetylglucosaminephosphotransferase 1 (GlcNAc-1-P transferase) (SEQ ID NO: 9985) and wherein said modified mRNA comprises modifications selected from the group consisting of:
(a) 5-methylcytosine and 2-thiouridine, (b) pseudouridine and 5-methylcytosine, and (c) 1-methylpseudouridine. |
| 地址 |
Cambridge MA US |
