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Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
专利权人:
GENEPOD THERAPEUTICS AB
发明人:
BJORKLUND, TOMAS,BJORKLUND, ANDERS,KIRIK, DENIZ
申请号:
NZ60010410
公开号:
NZ600104A
申请日:
2010.11.09
申请国别(地区):
NZ
年份:
2013
代理人:
摘要:
600104 Disclosed is a one-vector expression system comprising: a first and a second expression cassette, wherein said first expression cassette comprises a nucleotide sequence comprising a first promoter sequence operably linked to a first nucleotide sequence, said first nucleotide sequence encoding a GTP cyclohydrolase 1 (GCH1; EC 3.5.4.16) polypeptide or a biologically active fragment or variant thereof, and wherein said second expression cassette comprises a nucleotide sequence comprising a second promoter sequence operably linked to a second nucleotide sequence said second nucleotide sequence encoding a tyrosine hydroxylase (TH; EC 1.14.16.2) polypeptide or a biologically active fragment or variant thereof, and wherein the vector is an adeno associated vector (AAV) and wherein said second nucleotide sequence is operably linked to a Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE), and wherein both said first and said second promoter are Synapsin1 promoters.
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中国工程科技知识中心
来源网址:
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