| 发明名称 |
COMPOSITIONS AND METHODS FOR THE TREATMENT OF AADC DEFICIENCY |
| 摘要 |
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment, prophylaxis, palliation or amelioration of diseases, conditions and/or symptoms resulting from AADC deficiency and related inborn errors of neurotransmitter metabolism. |
| 申请公布号 |
WO2017023724(A1) |
申请公布日期 |
2017.02.09 |
| 申请号 |
WO2016US44627 |
申请日期 |
2016.07.29 |
| 申请人 |
VOYAGER THERAPEUTICS, INC. |
发明人 |
KOTIN, Robert;KELLS, Adrian Philip;RAVINA, Bernard |
| 分类号 |
A61K31/7088;A61K48/00;A61P25/26;C12N15/86 |
主分类号 |
A61K31/7088 |
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