Бьёрклунд Томас (SE),Бьёрклунд Андерс (SE),КИРИК Дениз (SE)
申请号：
RU2012123943/10
公开号：
RU2012123943A
申请日:
2010.11.09
申请国别(地区):
RU
年份:
2013
代理人:
摘要：
1. A one-way expression system comprising: first and second expression cassettes, said first expression cassette comprising a nucleotide sequence containing a first promoter sequence operably linked to a first nucleotide sequence, wherein the first nucleotide sequence encodes a GTP cyclohydrolase 1 polypeptide (GCH1； EC 3.5 .4.16) or a biologically active fragment or variant thereof, and wherein said second expression cassette contains a nucleotide sequence containing a promoter sequence operably linked to a second nucleotide sequence, said second nucleotide sequence encoding a tyrosine hydroxylase polypeptide (TH； EC 1.14.16.2) or a biologically active fragment or variant thereof, and the vector is an adeno-associated vector (AAV), and the second nucleotide sequence is functionally associated with the post-transcriptional regulatory element of marmot hepatitis virus (WPRE), and moreover, both the first and the second promo Ora is a promoter synapsin 1.2. The one-vector expression system according to claim 1, wherein the GTP cyclohydrolase 1 polypeptide (GCH1； EC 3.5.4.16) or a biologically active fragment or variant thereof is at least 70% identical to the polypeptide selected from the group consisting of SEQ ID NO. 1, SEQ ID NO. 2, SEQ ID NO. 3, SEQ ID NO. 4, SEQ ID NO. 5 and SEQ ID NO. 6, and wherein said tyrosine hydroxylase polypeptide (TH； EC 1.14.16.2) or a biologically active fragment or variant thereof is at least 70% identical to the polypeptide selected from the group consisting of SEQ ID NO. 7, SEQ ID NO. 8, SEQ ID NO. 9, SEQ ID NO. 10, SEQ ID NO. 11, SEQ ID1. Одновекторная система экспрессии, содержащая:первую и вторую кассеты экспрессии,причем указанная первая кассета экспрессии содержит нуклеотидную последовательность, содержащую первую промоторную последовательность, функционально связанную с первой нуклеотидной последовательностью,причем первая нуклеотидная последовательность кодирует полипептид GTP-циклогидролазы 1 (GCH1； ЕС 3.5.4.16) или его биоло
