The current perspective and opportunities of small nucleic acid-based therapeutics
作者全名："Chen, Yang; Li, Yang; Li, Chao; Zhang, Dandan; Liu, Yuheng; Zhang, Jingjing; Guan, Shan; Ding, Xiaoyan; Xiao, Qin"
作者地址："[Chen, Yang; Li, Chao; Zhang, Dandan; Liu, Yuheng; Zhang, Jingjing; Guan, Shan; Xiao, Qin] Third Mil Med Univ, Natl Engn Res Ctr Immunol Prod, Dept Microbiol & Biochem Pharm, Chongqing, Peoples R China; [Li, Yang] Third Mil Med Univ, Southwest Hosp, Dept Pharm, Chongqing, Peoples R China; [Liu, Yuheng] Chongqing Med Univ, Coll Pharm, Dept Pharmacol, Chongqing, Peoples R China; [Ding, Xiaoyan] Ludwig Maximilians Univ Munchen, Dept Pediat, Munich, Germany; [Xiao, Qin] Third Mil Med Univ, Natl Engn Res Ctr Immunol Prod, Dept Microbiol & Biochem Pharm, Chongqing 400038, Peoples R China; [Ding, Xiaoyan] Ludwig Maximilians Univ Munchen, Dept Pediat, D-80337 Munich, Germany"
通信作者："Xiao, Q (通讯作者)，Third Mil Med Univ, Natl Engn Res Ctr Immunol Prod, Dept Microbiol & Biochem Pharm, Chongqing 400038, Peoples R China.; Ding, XY (通讯作者)，Ludwig Maximilians Univ Munchen, Dept Pediat, D-80337 Munich, Germany."
来源：DRUG DEVELOPMENT RESEARCH
ESI学科分类：PHARMACOLOGY & TOXICOLOGY
WOS号：WOS:001173594900001
JCR分区：Q1
影响因子：3.8
年份：2024
卷号：85
期号：2
开始页：
结束页：
文献类型：Review
关键词：ASO; Patisiran; RNA therapeutic; small nucleic acid; siRNA
摘要："Compared to traditional small molecule and antibody drugs, RNA-based drugs offer a simple design, short research and development cycles, high specificity, broad treatment fields, and long-term efficacy. As a result, RNA-based drugs are extensively used to treat genetic diseases, tumors, viral infections, and other illnesses, suggesting that they have the potential to become the third-largest drug class after small molecule and antibody drugs. Currently, more than 10 small nucleic acid drugs have gained regulatory approval. The commercialization successes of small nucleic acid drugs will stimulate the development of RNA-based drugs. Small nucleic acid drugs primarily target liver diseases, metabolic diseases, genetic diseases, and tumors, and there is also significant potential for expanding indications in the future. This review provides a brief overview of the advantages and development of small nucleic acid-based therapeutics and shows a focus on platform technologies such as chemical modifications and delivery systems that have enabled the clinical translation of small nucleic acid-based therapeutics. Additionally, we summarize the latest clinical progress in small nucleic acid-based therapeutics for the treatment of various diseases, including rare diseases, liver diseases, metabolic diseases, and tumors. Finally, we highlight the future prospects for this promising treatment approach."
基金机构："Major project of Study on Pathogenesis and Epidemic Prevention Technology System [82173764, 2021YFC2302500]; National Natural Science Foundation of China; Ministry of Science and Technology of China [CQYC202005027]; Chongqing Talents: Exceptional Young Talents Project [cstc2021jcyj-msxmX0136]; Natural Science Foundation of Chongqing"
基金资助正文："This work was supported by the National Natural Science Foundation of China (Grant No. 82173764), the major project of Study on Pathogenesis and Epidemic Prevention Technology System (2021YFC2302500) by the Ministry of Science and Technology of China, the Chongqing Talents: Exceptional Young Talents Project (CQYC202005027) and the Natural Science Foundation of Chongqing (cstc2021jcyj-msxmX0136)."
