RNA interference in the era of nucleic acid therapeutics.
Journal
Nature biotechnology
ISSN: 1546-1696
Titre abrégé: Nat Biotechnol
Pays: United States
ID NLM: 9604648
Informations de publication
Date de publication:
Mar 2024
Mar 2024
Historique:
received:
05
10
2022
accepted:
15
12
2023
medline:
18
3
2024
pubmed:
27
2
2024
entrez:
27
2
2024
Statut:
ppublish
Résumé
Two decades of research on RNA interference (RNAi) have transformed a breakthrough discovery in biology into a robust platform for a new class of medicines that modulate mRNA expression. Here we provide an overview of the trajectory of small-interfering RNA (siRNA) drug development, including the first approval in 2018 of a liver-targeted siRNA interference (RNAi) therapeutic in lipid nanoparticles and subsequent approvals of five more RNAi drugs, which used metabolically stable siRNAs combined with N-acetylgalactosamine ligands for conjugate-based liver delivery. We also consider the remaining challenges in the field, such as delivery to muscle, brain and other extrahepatic organs. Today's RNAi therapeutics exhibit high specificity, potency and durability, and are transitioning from applications in rare diseases to widespread, chronic conditions.
Identifiants
pubmed: 38409587
doi: 10.1038/s41587-023-02105-y
pii: 10.1038/s41587-023-02105-y
doi:
Substances chimiques
RNA, Small Interfering
0
Acetylgalactosamine
KM15WK8O5T
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
394-405Informations de copyright
© 2024. Springer Nature America, Inc.
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