Therapeutic RNA-silencing oligonucleotides in metabolic diseases.
Journal
Nature reviews. Drug discovery
ISSN: 1474-1784
Titre abrégé: Nat Rev Drug Discov
Pays: England
ID NLM: 101124171
Informations de publication
Date de publication:
06 2022
06 2022
Historique:
accepted:
19
01
2022
pubmed:
26
2
2022
medline:
9
6
2022
entrez:
25
2
2022
Statut:
ppublish
Résumé
Recent years have seen unprecedented activity in the development of RNA-silencing oligonucleotide therapeutics for metabolic diseases. Improved oligonucleotide design and optimization of synthetic nucleic acid chemistry, in combination with the development of highly selective and efficient conjugate delivery technology platforms, have established and validated oligonucleotides as a new class of drugs. To date, there are five marketed oligonucleotide therapies, with many more in clinical studies, for both rare and common liver-driven metabolic diseases. Here, we provide an overview of recent developments in the field of oligonucleotide therapeutics in metabolism, review past and current clinical trials, and discuss ongoing challenges and possible future developments.
Identifiants
pubmed: 35210608
doi: 10.1038/s41573-022-00407-5
pii: 10.1038/s41573-022-00407-5
doi:
Substances chimiques
Oligonucleotides
0
Oligonucleotides, Antisense
0
RNA
63231-63-0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
417-439Informations de copyright
© 2022. Springer Nature Limited.
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