Gene‐based therapy is one of essential therapeutic strategies for precision medicine through targeting specific genes in specific cells of target tissues. However, there still exist many problems that need to be solved, such as safety, stability, selectivity, delivery, as well as immunity. Currently, the key challenges of gene‐based therapy for clinical potential applications are the safe and effective nucleic acid drugs as well as their safe and efficient gene delivery systems. In this review, we first focus on current nucleic acid drugs and their formulation in clinical trials and on the market, including antisense oligonucleotide, siRNA, aptamer, and plasmid nucleic acid drugs. Subsequently, we summarize different chemical modifications of nucleic acid drugs as well as their delivery systems for gene‐based therapeutics in vivo based on nucleic acid chemistry and nanotechnology methods.

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核酸药物的化学修饰及其基于基因治疗的递送系统

通过靶向靶组织特定细胞中的特定基因，基于基因的疗法是精准医学必不可少的治疗策略之一。但是，仍然存在许多需要解决的问题，例如安全性，稳定性，选择性，传递性和免疫性。当前，基于基因疗法在临床上的潜在应用面临的主要挑战是安全有效的核酸药物及其安全有效的基因递送系统。在这篇综述中，我们首先关注当前核酸药物及其在临床试验和市场上的制剂，包括反义寡核苷酸，siRNA，适体和质粒核酸药物。随后，