Therapeutic Mechanism of Nucleic Acid Drugs

Abstract Nucleic acid drugs were a class of DNA/RNA molecules and considered able to be applied for protein‐related diseases treatment through regulating target proteins expression. A number of nucleic acid drugs have been approved for treating various disease, caused by genetic defects, viral infections and genetic mutations. Based on different structural characteristics, this review provided a comprehensive overview about the nucleic acid drug's medicinal mechanism, clinical development, application, and also discussed the opportunities and challenges in nucleic acid drugs development.