Madrid Laura, Pinto-Díez Celia, Muñoz-Moreno Laura, García-Hernández Marta, González Víctor M, Mata Javier de la, Martin M Elena, Ortega Paula
Universidad de Alcalá, Department of Organic and Inorganic Chemistry, Research Institute in Chemistry "Andrés M. del Río" (IQAR), Chamartín, Madrid, 28805, Spain.
Networking Research Center on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), Madrid, 28029, Spain.
Macromol Biosci. 2025 Jan;25(1):e2400327. doi: 10.1002/mabi.202400327. Epub 2024 Oct 14.
Transfection efficiency is a critical parameter in gene therapy and molecular biology, representing the success rate at which nucleic acids are introduced and expressed in target cells. The combination of aptamers with nanotechnology-based delivery systems has demonstrated remarkable improvements in the transfection efficiency of therapeutic agents and holds significant potential for advancing gene therapy and the development of targeted treatments for various diseases, including cancer. In this work, cationic carbosilane dendritic systems are presented as an alternative to commercial transfection agents, demonstrating an increase in transfection efficiency when used for the internalization of apMNKQ2, an aptamer selected against a target in cancer. Their potential therapeutic use has been evaluated in breast cancer cell lines, MDA-MB-468 and MDA-MB-231, studying the cytotoxicity of the nanoconjugate, the internalization process, and its effect on cellular migration processes.
转染效率是基因治疗和分子生物学中的一个关键参数,代表核酸在靶细胞中导入和表达的成功率。适配体与基于纳米技术的递送系统相结合,已在治疗剂的转染效率方面取得了显著提高,并在推进基因治疗以及开发包括癌症在内的各种疾病的靶向治疗方面具有巨大潜力。在这项工作中,阳离子碳硅烷树枝状系统被作为商业转染剂的替代品提出,当用于内化apMNKQ2(一种针对癌症靶点筛选出的适配体)时,其转染效率有所提高。已在乳腺癌细胞系MDA-MB-468和MDA-MB-231中评估了它们的潜在治疗用途,研究了纳米缀合物的细胞毒性、内化过程及其对细胞迁移过程的影响。