发明名称 |
invivoでのニューロン特異的な最適化された連続DOPA合成用の新規ウイルスベクター構築物 |
摘要 |
The present invention relates to a one-vector expression system comprising a sequence encoding two polypeptides, such as tyrosine hydroxylase (TH) and GTP-cyclohydrolase 1 (GCH1). The two polypeptides can be should preferentially be expressed at a ratio between 3:1 and 15:1, such as between 3:1 and 7:1. The invention is useful in the treatment of catecholamine deficient disorders, such as dopamine deficient disorders including but not limited to Parkinson's Disease. Moreover, the present invention provides a method to deliver the vector construct in order to limit the increased production of the catecholamine to the cells in need thereof. |
申请公布号 |
JP5894535(B2) |
申请公布日期 |
2016.03.30 |
申请号 |
JP20120538313 |
申请日期 |
2010.11.09 |
申请人 |
ジーンポッド セラピューティクス アーベーGENEPOD THERAPEUTICS AB |
发明人 |
ビョルクルンド,トマス;ビョルクルンド,アンデシュ;キリク,デニス |
分类号 |
C12N15/09;A61K35/76;A61K38/36;A61K48/00;A61P9/12;A61P13/12;A61P25/00;A61P25/02;A61P25/16;A61P25/18;A61P25/28;A61P25/32;A61P25/36;C12N1/15;C12N1/19;C12N5/10;C12Q1/26;C12Q1/34 |
主分类号 |
C12N15/09 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|