公开(公告)号：US20080207539A1

公开(公告)日：2008-08-28

申请号：US10568933

申请日：2004-08-31

申请人： Patrick Arbuthnot ,  Marc Weinberg ,  Sergio Catril Carmona ,  Ely Abdullah

发明人： Patrick Arbuthnot ,  Marc Weinberg ,  Sergio Catril Carmona ,  Ely Abdullah

IPC分类号： A61K31/70 ,  C12N15/00 ,  C12N5/00 ,  C07H21/04

CPC分类号： C12N15/111 ,  A61K48/00 ,  C12N15/1131 ,  C12N2310/121 ,  C12N2310/123 ,  C12N2310/128 ,  C12N2310/14 ,  C12N2310/51 ,  C12N2330/50 ,  C12N2310/531

摘要： The invention provides a self-processing RNA expression cassette which includes at least one pair of processing units, an RNAi effecter sequence of predetermined length that regulates target gene expression which is flanked by said pair of processing units; and at least one pair of cognate ribozyme cis-cleavage target sites located 5′ and 3′ of the RNAi effecter sequence. The self-processing RNA expression cassette is able to express in vivo and in vitro and the RNAi effecter sequence includes at least one target recognition sequence derived from the Hepatitis B Virus (HBV) X gene (HBx).

摘要翻译： 本发明提供了一种自我处理的RNA表达盒，其包括至少一对处理单元，调节靶基因表达的预定长度的RNAi效应子序列，其侧翼是所述一对处理单元; 以及位于RNAi效应子序列5'和3'的至少一对同源核酶顺式裂解靶位点。 自身处理的RNA表达盒能够在体内和体外表达，并且RNAi效应物序列包括至少一种源自乙型肝炎病毒（HBV）X基因（HBx）的目标识别序列。