| 发明名称 |
MODIFIED POLYNUCLEOTIDES FOR TREATING GALACTOSIDASE, ALPHA 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 |
US2014200263(A1) |
申请公布日期 |
2014.07.17 |
| 申请号 |
US201414170751 |
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 |
C12N15/52;C12N9/24 |
主分类号 |
C12N15/52 |
| 代理机构 |
|
代理人 |
|
| 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 galactosidase, alpha (SEQ ID NO: 998), 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 |
