Progress in research on mRNA drugs for the treatment of genetic diseases
10.3760/cma.j.cn511374-20230203-00051
- VernacularTitle:mRNA药物在遗传性疾病治疗领域的研究进展
- Author:
Wenjun SHI
1
;
Xianshuo CHENG
;
Pei LUO
;
Jilong MA
;
Jian DONG
Author Information
1. 云南省第一人民医院/昆明理工大学附属医院肿瘤内科，昆明 650032
- Keywords:
mRNA therapeutics;
Genetic disorder;
In vitro transcription
- From:
Chinese Journal of Medical Genetics
2024;41(3):368-376
- CountryChina
- Language:Chinese
-
Abstract:
In recent years, mRNA drugs have shown a great potential for the treatment of genetic diseases and attracted the attention of many researchers. This article has reviewed the advance in the research of mRNA drugs for the treatment of genetic diseases over the past 30 years, including their mechanisms of action and structure design, with a focus on their advantages as alternative therapies such as high specificity, low dosage, and sustained expression. Meanwhile, challenges for the effective delivery and storage methods for the mRNA drugs are discussed, with an aim to provide guidance for subsequent researches.
