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BICISTRONIC AAV VECTOR FOR RNA INTERFERENCE IN ALS
专利权人:
发明人:
Julianne Aebischer,Bernard Schneider,Cylia Rochat
申请号:
US15746458
公开号:
US20180228919A1
申请日:
2016.07.22
申请国别(地区):
US
年份:
2018
代理人:
摘要:
The present invention relates to a bicistronic expression vector for silencing a gene specifically in astrocytes and neurons, comprising two expression cassettes comprising a first and a second silencer sequence, respectively, wherein the expression of said first silencer sequence within astrocytes is regulated by an astrocyte-specific promoter and the expression of said second silencer sequence within neurons is regulated by a neuron-specific promoter. In a preferred embodiment, said first and second silencer sequences are SOD1 silencer sequences. Pharmaceutical composition comprising said bicistronic vector and the use of the same in the treatment of motoneuron diseases are further described.
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