The delivery of nucleic acids with transient activity for genetic engineering is a promising methodology with potential applications in the treatment of diseases ranging from cancer and infectious diseases to heritable disorders. Restoring the expression of a missing protein, correcting defective splicing of transcripts and silencing or modulating the expression of genes are powerful approaches that could have substantial benefits in biological research and medicine. Impressive progress in improving gene delivery has been made in the past decade, and several products have reached the market. However, translating the results of in vitro and preclinical studies into functional therapies is hindered by the suboptimal performance of gene delivery vehicles in capturing, protecting and delivering nucleic acid cargoes safely and efficaciously. Chemistry has a key role in the development of innovative synthetic materials to overcome the challenges of producing next-generation gene delivery therapies and protocols. In this Review, we discuss the latest chemical advances in the production of materials for the delivery of nucleic acids to cells and for gene therapy.

用于基因工程的具有瞬时活性的核酸的递送是一种有前途的方法，其在治疗从癌症和传染性疾病到遗传性疾病的疾病中具有潜在的应用。恢复丢失的蛋白质的表达，纠正转录物的有缺陷的剪接，沉默或调节基因的表达是有效的方法，可以在生物学研究和医学中带来实质性的好处。在过去的十年中，在改善基因传递方面取得了令人印象深刻的进步，并且有几种产品进入了市场。然而，将基因递送载体在安全有效地捕获，保护和递送核酸货物中的次优性能阻碍了将体外和临床前研究的结果转化为功能疗法的过程。化学在创新合成材料的开发中起着关键作用，以克服产生下一代基因传递疗法和方案的挑战。在这篇综述中，我们讨论了用于将核酸递送至细胞和用于基因治疗的材料生产中的最新化学进展。