发明名称 Tissue-specific and target rna-specific ribozymes
摘要 The present invention relates to multi-ribozymes and their use to target RNA in a tissue-specific, target RNA-specific, or pathogen-specific manner for the treatment of cancers, proliferative disease, and bacterial, parasitic and viral infections. More specifically, the present invention relates to the use of virions and viral vectors to package and deliver DNA encoding the multi-ribozymes to a host. The present invention relates to the use of liposomes and lipid-DNA complexes to deliver DNA encoding ribozymes to a host. Most specifically, the invention relates to the use of target specific virions to package and deliver DNA comprising a target specific promoter and encoding a ribozyme(s) directed to the target organism nucleic acids. The present invention further relates to a novel vectors encoding a multi-ribozyme structure with enhanced 5′ and/or 3′ autocatalytically cleaving ribozymes. The invention further relates to nucleotides encoding a multi-ribozyme comprising one or more ribozyme cassettes which contain one or more trans-acting ribozymes and one or more autocatalytically cleaving ribozyme sequences.
申请公布号 AU4830899(A) 申请公布日期 2000.01.10
申请号 AU19990048308 申请日期 1999.06.24
申请人 MUSC FOUNDATION FOR RESEARCH DEVELOPMENT;THE PENN STATE RESEARCH FOUNDATION 发明人 JAMES S. NORRIS;GARY A. CLAWSON;MICHAEL G SCHMIDT;BRIAN HOEL;WEIHUA PAN;JOE DOLAN;DAVID SCHOLFIELD;CANCAN HUANG
分类号 A61K38/00;C12N15/113 主分类号 A61K38/00
代理机构 代理人
主权项
地址