发明名称 |
METHODS AND COMPOSITIONS FOR EXPRESSING HETEROLOGOUS GENES IN HEPATOCYTES USING HEPADNAVIRAL VECTORS |
摘要 |
Methods and compositions for efficient, hepatocyte-specific delivery and expression of heterologous gens, both in vitro and in vivo, using hepadnaviral vectors are provided. Methods for expressing a heterologous gene in hepatocytes are provided involving: providing replication defective hepadnavirus particles at a titre level competent to infect hepatocytes, wherein a region of the preS/S-gene of the hepadnavirus genome has been replaced with the heterologous gene such that expression of the heterologous gene is regulated by regulatory sequences of the preS/S-gene; and infecting hapatocytes with the hepadnavirus such that the heterologous gene is delivered into the hepatocytes and expressed in the hepatocytes. Methods for treating a subject with a hepatic disorder (e.g., hepatitis infection) are also provided. Replication defective hepadnavirus particles, and pharmaceutical compositions thereof, are also provided. Methods of producing therapeutic replication defective hepadnavirus particles at a titre level suitable for therapeutic use are also provided.
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申请公布号 |
WO0012739(A1) |
申请公布日期 |
2000.03.09 |
申请号 |
WO1999US19452 |
申请日期 |
1999.08.26 |
申请人 |
BASF AKTIENGESELLSCHAFT;SCHALLER, HEINZ;PROTZER, ULRIKE;NASSAL, MICHAEL |
发明人 |
SCHALLER, HEINZ;PROTZER, ULRIKE;NASSAL, MICHAEL |
分类号 |
A61P1/16;A61P35/00;C12N7/01;C12N15/86;(IPC1-7):C12N15/86;A61K48/00 |
主分类号 |
A61P1/16 |
代理机构 |
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地址 |
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