Gene therapy requires successful delivery of therapeutic nucleic acids into target cells; thus, efficient and safe gene delivery carriers are crucial to its success. Although many polymeric materials have shown their potential as effective nucleic acid carriers, the inherent heterogeneity and polydispersity of these polymers hinder their application in clinical studies because of difficulties in their further precise modification, structure–activity relationship study, as well as consistent manufacturing. Therefore, precisely defined polymers, with potential for site-specific optimization according to structure–activity relationship information and highly controllable production, have been extensively investigated. In this review, we focus on the design and development of precisely defined polymers for efficient gene delivery, illustrated with examples including dendrimers, peptide-based polymers, and sequence-defined oligoaminoamide oligomers.

基因治疗需要将治疗性核酸成功递送到靶细胞中。因此，高效，安全的基因传递载体对其成功至关重要。尽管许多聚合物材料已显示出它们可作为有效的核酸载体的潜力，但这些聚合物固有的异质性和多分散性阻碍了它们在临床研究中的应用，因为它们难以进行进一步的精确修饰，结构-活性关系研究以及稳定的生产。因此，已经对广泛定义的聚合物进行了广泛研究，这些聚合物具有根据结构-活性关系信息和高度可控的生产进行特定位置优化的潜力。在这篇综述中，我们专注于精确定义的聚合物的设计和开发，以有效地进行基因传递，