申请人:Davis E. Mark
公开号:US20050136430A1
公开(公告)日:2005-06-23
The present invention provides methods and compositions for attenuating expression of a target gene in vivo. In general, the method includes administering RNAi constructs (such as small-interfering RNAs (i.e., siRNAs) that are targeted to particular mRNA sequences, or nucleic acid material that can produce siRNAs in a cell), in an amount sufficient to attenuate expression of a target gene by an RNA interference mechanism. In particular, the RNAi constructs include one or more modifications to improve serum stability and cellular uptake and to avoid non-specific effect.
本发明提供了减弱体内靶基因表达的方法和组合物。 一般来说,该方法包括施用RNAi构建物(如针对特定mRNA序列的小干扰RNA(即siRNA),或可在细胞中产生siRNA的核酸材料),其用量足以通过RNA干扰机制减弱靶基因的表达。 特别是,RNAi 构建物包括一种或多种修饰,以提高血清稳定性和细胞吸收率,并避免非特异性效应。