| 发明名称 |
MRNA THERAPY FOR ARGININOSUCCINATE SYNTHETASE DEFICIENCY |
| 摘要 |
The present invention provides, among other things, methods of treating Argininosuccinate Synthetase Deficiency (ASD), including administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids |
| 申请公布号 |
US2015110859(A1) |
申请公布日期 |
2015.04.23 |
| 申请号 |
US201414521351 |
申请日期 |
2014.10.22 |
| 申请人 |
Shire Human Genetic Therapies, Inc. |
发明人 |
Heartlein Michael;DeRosa Frank;Smith Lianne |
| 分类号 |
A61K38/53;A61K9/127;C12N9/00 |
主分类号 |
A61K38/53 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method of treating Argininosuccinate Synthetase Deficiency (ASD), comprising administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset. |
| 地址 |
Lexington MA US |
