| 发明名称 |
MODIFIED POLYNUCLEOTIDES FOR TREATING ARGININOSUCCINATE SYNTHASE 1 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 |
US2014155472(A1) |
申请公布日期 |
2014.06.05 |
| 申请号 |
US201414170897 |
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 |
C12N9/00 |
主分类号 |
C12N9/00 |
| 代理机构 |
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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 a member selected from the group consisting of argininosuccinate synthase 1 (SEQ ID NO: 830), argininosuccinate synthase 1 (SEQ ID NO: 831), argininosuccinate synthase 1 (SEQ ID NO: 832), argininosuccinate synthase 1 (SEQ ID NO: 833), argininosuccinate synthase 1 (SEQ ID NO: 834), argininosuccinate synthase 1 (SEQ ID NO: 835) and argininosuccinate synthase 1 (SEQ ID NO: 836), 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.
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| 地址 |
CAMBRIDGE MA US |
