RNA-based medicine: from molecular mechanisms to therapy.
CRISPR-Cas therapy
RNA interference
antisense oligonucleotide drug
mRNA therapeutics
rare genetic disease
Journal
The EMBO journal
ISSN: 1460-2075
Titre abrégé: EMBO J
Pays: England
ID NLM: 8208664
Informations de publication
Date de publication:
02 11 2023
02 11 2023
Historique:
revised:
24
08
2023
received:
14
06
2023
accepted:
29
08
2023
medline:
3
11
2023
pubmed:
20
9
2023
entrez:
20
9
2023
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 37728251
doi: 10.15252/embj.2023114760
pmc: PMC10620767
doi:
Substances chimiques
RNA
63231-63-0
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e114760Informations de copyright
© 2023 The Authors. Published under the terms of the CC BY 4.0 license.
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