Aix-Marseille Université, Center Interdisciplinaire de Nanoscience de Marseille, UMR 7325, Equipe Labellisée Ligue Contre le Cancer, CNRS, Marseille, France.
State Key Laboratory of Natural Medicines and Jiangsu Key Laboratory of Drug Discovery for Metabolic Diseases, Center of Drug Discovery, Center of Advanced Pharmaceutics and Biomaterials, China Pharmaceutical University, Nanjing, P. R. China.
Nat Protoc. 2021 Jan;16(1):327-351. doi: 10.1038/s41596-020-00418-9. Epub 2020 Dec 4.
Using siRNAs to genetically manipulate immune cells is important to both basic immunological studies and therapeutic applications. However, siRNA delivery is challenging because primary immune cells are often sensitive to the delivery materials and generate immune responses. We have recently developed an amphiphilic dendrimer that is able to deliver siRNA to a variety of cells, including primary immune cells. We provide here a protocol for the synthesis of this dendrimer, as well as siRNA delivery to immune cells such as primary T and B cells, natural killer cells, macrophages, and primary microglia. The dendrimer synthesis entails straightforward click coupling followed by an amidation reaction, and the siRNA delivery protocol requires simple mixing of the siRNA and dendrimer in buffer, with subsequent application to the primary immune cells to achieve effective and functional siRNA delivery. This dendrimer-mediated siRNA delivery largely outperforms the standard electroporation technique, opening a new avenue for functional and therapeutic studies of the immune system. The whole protocol encompasses the dendrimer synthesis, which takes 10 days; the primary immune cell preparation, which takes 3-10 d, depending on the tissue source and cell type; the dendrimer-mediated siRNA delivery; and subsequent functional assays, which take an additional 3-6 d.
使用 siRNA 对免疫细胞进行基因操作对于基础免疫学研究和治疗应用都很重要。然而,siRNA 的递送具有挑战性,因为原代免疫细胞通常对递送材料敏感并产生免疫反应。我们最近开发了一种两亲性树状聚合物,能够将 siRNA 递送至各种细胞,包括原代免疫细胞。这里我们提供了该树状聚合物的合成方案,以及将 siRNA 递送至免疫细胞(如原代 T 细胞和 B 细胞、自然杀伤细胞、巨噬细胞和原代小神经胶质细胞)的方法。树状聚合物的合成需要简单的点击偶联反应,然后进行酰胺化反应,siRNA 递送方案只需将 siRNA 和树状聚合物在缓冲液中简单混合,然后应用于原代免疫细胞,即可实现有效的功能性 siRNA 递送。这种树状聚合物介导的 siRNA 递送在很大程度上优于标准的电穿孔技术,为免疫系统的功能和治疗研究开辟了新途径。整个方案包括树状聚合物的合成,需要 10 天;原代免疫细胞的制备,根据组织来源和细胞类型,需要 3-10 天;树状聚合物介导的 siRNA 递送;以及后续的功能测定,需要再额外 3-6 天。