Abstract: 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.
Type:
Grant
Filed:
April 1, 2014
Date of Patent:
March 14, 2017
Assignee:
Genepod Therapeutics AB
Inventors:
Tomas Björklund, Anders Björklund, Deniz Kirik
Abstract: 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.
Type:
Application
Filed:
November 9, 2010
Publication date:
December 6, 2012
Applicant:
GENEPOD THERAPEUTICS AB
Inventors:
Tomas Björklund, Anders Björklund, Deniz Kirik