公开(公告)号：US20240115737A1

公开(公告)日：2024-04-11

申请号：US18277023

申请日：2022-02-14

申请人： UCL Business Ltd

发明人： Joanna NG ,  Manju KURIAN ,  Simon WADDINGTON ,  John COUNSELL

IPC分类号： A61K48/00 ,  A01K67/0276 ,  A61K9/00 ,  A61P25/28 ,  C07K14/705 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A01K67/0276 ,  A61K9/0019 ,  A61K9/0085 ,  A61K48/0075 ,  A61K48/0083 ,  A61P25/28 ,  C07K14/70571 ,  C12N15/86 ,  C12N2750/14143

摘要： There is described a vector for treating Dopamine Transporter Deficiency Syndrome, the vector comprising a promoter operably linked to a human SLC6A3 gene, wherein the promoter is selected from a human synapsin 1 promoter, a CAG promoter, a CMV promoter, a CAMKII promoter, a beta-actin promoter, and a human EF1-alpha promoter. Also described are methods and uses involving the vector for treating Dopamine Transporter Deficiency Syndrome.