Therapeutic Oligonucleotides: An Outlook on Chemical Strategies to Improve Endosomal Trafficking.
antisense oligonucleotides (ASOs)
cleavable linkers
endosomal escape
endosomolytic agents
linker chemistry
non-cleavable linkers
oligonucleotide conjugates
oligonucleotide delivery
oligonucleotide therapeutics
pH-sensitive linkers
siRNA
Journal
Cells
ISSN: 2073-4409
Titre abrégé: Cells
Pays: Switzerland
ID NLM: 101600052
Informations de publication
Date de publication:
11 09 2023
11 09 2023
Historique:
received:
21
06
2023
revised:
30
08
2023
accepted:
07
09
2023
medline:
29
9
2023
pubmed:
28
9
2023
entrez:
28
9
2023
Statut:
epublish
Résumé
The potential of oligonucleotide therapeutics is undeniable as more than 15 drugs have been approved to treat various diseases in the liver, central nervous system (CNS), and muscles. However, achieving effective delivery of oligonucleotide therapeutics to specific tissues still remains a major challenge, limiting their widespread use. Chemical modifications play a crucial role to overcome biological barriers to enable efficient oligonucleotide delivery to the tissues/cells of interest. They provide oligonucleotide metabolic stability and confer favourable pharmacokinetic/pharmacodynamic properties. This review focuses on the various chemical approaches implicated in mitigating the delivery problem of oligonucleotides and their limitations. It highlights the importance of linkers in designing oligonucleotide conjugates and discusses their potential role in escaping the endosomal barrier, a bottleneck in the development of oligonucleotide therapeutics.
Identifiants
pubmed: 37759475
pii: cells12182253
doi: 10.3390/cells12182253
pmc: PMC10527716
pii:
doi:
Substances chimiques
Oligonucleotides
0
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
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