Progress on RNA-based therapeutics for genetic diseases.
10.3724/zdxbyxb-2023-0190
- Author:
Ting LUO
1
;
Chunxiao HUO
2
;
Tianhua ZHOU
3
;
Shanshan XIE
4
Author Information
1. Department of Cell Biology, Zhejiang University School of Medicine, Hangzhou 310058, China. tingluo1117@163.com.
2. Department of Cell Biology, Zhejiang University School of Medicine, Hangzhou 310058, China.
3. Department of Cell Biology, Zhejiang University School of Medicine, Hangzhou 310058, China. tzhou@zju.edu.cn.
4. Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou 310052, China. sxie@zju.edu.cn.
- Publication Type:Journal Article
- Keywords:
Antisense oligonucleotides;
Genetic disease;
Messenger RNA drugs;
RNA aptamers;
RNA, drug;
RNA, medicine;
Review;
Small interfering RNA
- MeSH:
Aptamers, Nucleotide;
RNA, Small Interfering/therapeutic use*;
RNA, Messenger;
Oligonucleotides, Antisense/therapeutic use*
- From:
Journal of Zhejiang University. Medical sciences
2023;52(4):406-416
- CountryChina
- Language:English
-
Abstract:
RNA therapeutics inhibit the expression of specific proteins/RNAs by targeting complementary sequences of corresponding genes or encode proteins for the synthesis desired genes to treat genetic diseases. RNA-based therapeutics are categorized as oligonucleotide drugs (antisense oligonucleotides, small interfering RNA, RNA aptamers), and mRNA drugs. The antisense oligonucleotides and small interfering RNA for treatment of genetic diseases have been approved by the FDA in the United States, while RNA aptamers and mRNA drugs are still in clinical trials. Chemical modifications can be applied to RNA drugs, such as pseudouridine modification of mRNA, to reduce immunogenicity and improve the efficacy. The secure and effective delivery systems such as lipid-based nanoparticles, extracellular vesicles, and virus-like particles are under development to address stability, specificity, and safety issues of RNA drugs. This article provides an overview of the specific molecular mechanisms of eleven RNA drugs currently used for treating genetic diseases, and discusses the research progress of chemical modifications and delivery systems of RNA drugs.
