南方科技大学材料科学与工程学院田雷蕾研究组取得一项新突破，他们利用一种可基因编码和生物合成的DNA树枝状大分子实现了靶向基因调控。 这一研究成果发表在2020年11月23日出版的国际学术期刊《德国应用化学》上。

在该研究中，课题组设计了一种适用于生物生产的DNA树枝状大分子纳米药物，该分子由灵活的三臂单元组成以实现高效的一锅法DNA组装。该分子，即D4‐3‐As‐DzSur，是第一种实现基因编码、生物技术合成和直接自组装的DNA纳米药物。这种生物合成的D4‐3‐As‐DzSur在靶向基因调控中的表现在体外和体内实验中得到了证实。研究人员认为，可由生物生产是该分子的一大优势，这一特点使得低成本、大规模的全DNA纳米药物生产成为可能，在未来亦会大大促进其在临床中的应用。

据了解，全DNA纳米药物已成为一种潜在的新型抗肿瘤药物。DNA纳米技术为控制全DNA纳米药物的尺寸、形貌及其负载的药物分子的多元化提供了无限的可能性。更重要的是，由于DNA是一种生物高分子，这种全DNA纳米药物可在活细菌中实现基因编码和生产。

附：英文原文

Title: Genetically Encoded and Biologically Produced All‐DNA Nanomedicine Based on One‐Pot Assembly of DNA Dendrimers for Targeted Gene Regulation

Author: Jingxiong Lu, Pengchao Hu, Lingyan Cao, Zixiang Wei, Fan Xiao, Zhe Chen, Yan Li, Leilei Tian

Issue&Volume: 23 November 2020

Abstract: All‐DNA nanomedicines have emerged as potential anti‐tumor drugs. DNA nanotechnology provides all‐DNA nanomedicines unlimited possibilities in controlling the diversification of size, shape, and loads of the therapeutic motifs. What’s more, as DNA is a biological polymer, it is possible to genetically encode and produce the all‐DNA nanomedicines in living bacteria. Herein, DNA‐dendrimer‐based nanomedicines are designed to adapt to the biological production, which is constructed by the flexible 3‐arm building blocks to enable a highly efficient “one‐pot” DNA assembly. For the first time, a DNA nanomedicine, D4‐3‐As‐DzSur, is successfully genetically encoded, biotechnological produced, and directly self‐assembled. By the following  in vitro  and  in vivo  studies, the performance of the biologically produced D4‐3‐As‐DzSur in targeted gene regulation has been confirmed. We conclude that, as an advantageous superiority, the biological production capability will fulfill the low‐cost and large‐scale production of all‐DNA nanomedicines and promote their clinical applications in the future.

DOI: 10.1002/anie.202012916

Source: https://onlinelibrary.wiley.com/doi/10.1002/anie.202012916