Yamada Yoji
Research Management Office, Research Unit, R&D Division, Kyowa Kirin Co. Ltd., 1-9-2, Otemachi, Chiyoda-ku, Tokyo 100-0004, Japan.
Cancers (Basel). 2021 Oct 5;13(19):5002. doi: 10.3390/cancers13195002.
Nucleic acid drugs are being developed as novel therapeutic modalities. They have great potential to treat human diseases such as cancers, viral infections, and genetic disorders due to unique characteristics that make it possible to approach undruggable targets using classical small molecule or protein/antibody-based biologics. In this review, I describe the advantages, classification, and clinical status of nucleic acid therapeutics. To date, more than 10 products have been launched, and many products have been tested in clinics. To promote the use of nucleic acid therapeutics such as antibodies, several hurdles need to be surmounted. The most important issue is the delivery of nucleic acids and several other challenges have been reported. Recent advanced delivery platforms are lipid nanoparticles and ligand conjugation approaches. With the progress of exosome biology, exosomes are expected to contribute to the solution of various problems associated with nucleic acid drugs.
核酸药物正作为新型治疗方式进行研发。由于其独特的特性，使得利用传统小分子或基于蛋白质/抗体的生物制剂靶向难以成药的靶点成为可能，因此它们在治疗癌症、病毒感染和遗传疾病等人类疾病方面具有巨大潜力。在这篇综述中，我描述了核酸疗法的优势、分类和临床现状。迄今为止，已有10多种产品上市，许多产品已在临床试验中进行测试。为了促进核酸疗法（如抗体）的应用，需要克服几个障碍。最重要的问题是核酸的递送，并且已经报道了其他一些挑战。最近先进的递送平台是脂质纳米颗粒和配体偶联方法。随着外泌体生物学的进展，外泌体有望有助于解决与核酸药物相关的各种问题。
