TOMAS BJORKLUND;GENEPOD THERAPEUTICS AB;ANDERS BJORKLUND;DENIZ KIRIK
发明人:
申请号：
IL21970412
公开号：
IL219704D0
申请日:
2012.05.09
申请国别(地区):
IL
年份:
2012
代理人:
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.
