申请人:——
公开号:US20030166601A1
公开(公告)日:2003-09-04
Non-naturally occurring vector for gene therapy are provided, comprised of chemically defined reagents, where the vector is self-assembling and where the vector comprises (1) a core complex comprising a nucleic acid and (2) at least one complex forming reagent, where the vector has fusogenic activity. The vector optionally may contain reagents permitting fusion with cell membranes and nuclear uptake. The vector also may contain an outer shell moiety that is anchored to the core complex, whereby the outer shell stabilizes the complex, protects it from unwanted interactions and enhances delivery of the nucleic acid into a target tissue or cell. The outer shell optionally may be sheddable, that is, it may be designed such that it dissociates from the vector upon entry into the target cell or tissue.
提供了一种非自然发生的基因治疗载体,由化学定义的试剂组成,其中载体是自组装的,包括(1)核酸的核心复合物和(2)至少一个复合物形成试剂,其中载体具有融合活性。该载体可选地包含允许与细胞膜融合和核摄取的试剂。该载体还可以包含一个外壳基团,该基团固定在核心复合物上,从而稳定外壳,保护它免受不必要的相互作用,并增强核酸传递到目标组织或细胞的能力。外壳可选地可脱落,即它可以被设计成在进入目标细胞或组织时与载体分离。