Perspectives of genetic management strategy for inherited cardiovascular diseases in China

[...]there is an urgent need to develop better therapeutic strategies for treating inherited CVDs. Inclisiran (ALN-PCSsc), approved for the use in a clinical trial, is a long-acting RNA interference (RNAi) therapeutic agent that inhibits the synthesis of proprotein convertase subtilisin–kexin type 9...

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Bibliographic Details
Published in:Chinese medical journal 2024-04, Vol.137 (8), p.959-961
Main Authors: Zhang, Yaoyao, Zheng, Yanjiang, Dai, Mengyuan, Zhou, Kaiyu, Fu, Lijun, Guo, Yuxuan, He, Yihua, Li, Fen, Gao, Rui, Zhang, Donghui, Liu, Xujie, Tian, Jie, Hua, Yimin, Li, Yifei, Qin, Lang
Format: Article
Language:English
Subjects:
Cancer therapies
Cardiovascular disease
Cholesterol
Clinical trials
CRISPR
Disease prevention
Efficiency
Gene therapy
Genetic testing
Mutation
Pregnancy
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Summary:[...]there is an urgent need to develop better therapeutic strategies for treating inherited CVDs. Inclisiran (ALN-PCSsc), approved for the use in a clinical trial, is a long-acting RNA interference (RNAi) therapeutic agent that inhibits the synthesis of proprotein convertase subtilisin–kexin type 9 (Pcsk9), a target for lowering low-density lipoprotein (LDL) cholesterol to prevent coronary heart disease. [...]providing disease management for such a population is a great challenge. [...]preimplantation genetic testing (PGT) technology has been applied in the prevention of ICD, so that couples with genetic risk of ICD can avoid termination of pregnancy and give birth to healthy children without ICD susceptibility genes. [...]gene therapy is the most powerful and efficient method of managing inherited CVDs, but there is a long way to go to achieve this medical goal.
ISSN:0366-6999
2542-5641
DOI:10.1097/CM9.0000000000002804