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- W4386877877 abstract "RNA-based therapeutics have the potential to revolutionize the treatment and prevention of human diseases. While early research faced setbacks, it established the basis for breakthroughs in RNA-based drug design that culminated in the extraordinarily fast development of mRNA vaccines to combat the COVID-19 pandemic. We have now reached a pivotal moment where RNA medicines are poised to make a broad impact in the clinic. In this review, we present an overview of different RNA-based strategies to generate novel therapeutics, including antisense and RNAi-based mechanisms, mRNA-based approaches, and CRISPR-Cas-mediated genome editing. Using three rare genetic diseases as examples, we highlight the opportunities, but also the challenges to wide-ranging applications of this class of drugs." @default.
- W4386877877 created "2023-09-21" @default.
- W4386877877 creator A5037571507 @default.
- W4386877877 creator A5041877094 @default.
- W4386877877 date "2023-09-20" @default.
- W4386877877 modified "2023-09-27" @default.
- W4386877877 title "<scp>RNA</scp>‐based medicine: from molecular mechanisms to therapy" @default.
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