Antisense Oligonucleotides and Small Interfering RNA for the Treatment of Dyslipidemias.
ASO
hypercholesterolemia
miRNA
siRNAF
Journal
Journal of clinical medicine
ISSN: 2077-0383
Titre abrégé: J Clin Med
Pays: Switzerland
ID NLM: 101606588
Informations de publication
Date de publication:
04 Jul 2022
04 Jul 2022
Historique:
received:
06
04
2022
revised:
21
06
2022
accepted:
01
07
2022
entrez:
9
7
2022
pubmed:
10
7
2022
medline:
10
7
2022
Statut:
epublish
Résumé
The burden of atherosclerotic disease worldwide necessitates implementing the treatment of its risk factors. Among them, hypercholesterolemia has a central role. In addition to conventional small organic compounds and the recently introduced monoclonal antibodies, new technologies are arising such as the antisense oligonucleotides and small interfering RNAs (siRNAs) that operate upstream, blocking the mRNA translation of the proteins specifically involved in lipid metabolism. In this review, we briefly explain the mechanisms of action of these molecules and discuss the difficulties related to their in vivo use as therapeutical agents. We go over the oligonucleotides tested in clinical trials that could potentially revolutionize the care of patients by acting on proteins involved in the lipoprotein metabolism and regulation, namely: angiopoietin-like protein 3 (ANGPTL3); lipoprotein a (Lp(a)); apolipoprotein B (Apo B); apolipoprotein C III (Apo C-III); and proprotein convertase subtilisin-kexin type 9 (PCSK9). Finally, the differences between ASOs and siRNAs, their future possible clinical applications, and the role of Inclisiran, a siRNA direct against PCSK9 to reduce LDL-C, were reviewed in detail.
Identifiants
pubmed: 35807171
pii: jcm11133884
doi: 10.3390/jcm11133884
pmc: PMC9267663
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
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