申请人:Case Western Reserve University
公开号:US05972901A1
公开(公告)日:1999-10-26
Nucleic acids are compacted, substantially without aggregation, to facilitate their uptake by target cells of an organism to which the compacted material is administered. The nucleic acids may achieve a clinical effect as a result of gene expression, hybridization to endogenous nucleic acids whose expression is undesired, or site-specific integration so that a target gene is replaced, modified or deleted. The targeting may be enhanced by means of a target cell-binding moiety. The nucleic acid is preferably compacted to a condensed state.
核酸被压缩,实现无聚集,以便于其被注射到生物体的靶细胞中。这些核酸可能通过基因表达、杂交到不需要表达的内源性核酸或者位点特异性整合来实现临床效应,以替换、修改或删除目标基因。这种定向作用可以通过靶细胞结合基团来增强。核酸最好被压缩到一种紧凑的状态。