In:
The EMBO Journal, EMBO, Vol. 42, No. 21 ( 2023-11-02)
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.
Type of Medium:
Online Resource
ISSN:
0261-4189
,
1460-2075
DOI:
10.15252/embj.2023114760
Language:
English
Publisher:
EMBO
Publication Date:
2023
detail.hit.zdb_id:
1467419-1
detail.hit.zdb_id:
586044-1
SSG:
12
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