Advances in oligonucleotide drug delivery.
Nat Rev Drug Discov
; 19(10): 673-694, 2020 10.
Article
in En
| MEDLINE
| ID: mdl-32782413
ABSTRACT
Oligonucleotides can be used to modulate gene expression via a range of processes including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation, non-coding RNA inhibition, gene activation and programmed gene editing. As such, these molecules have potential therapeutic applications for myriad indications, with several oligonucleotide drugs recently gaining approval. However, despite recent technological advances, achieving efficient oligonucleotide delivery, particularly to extrahepatic tissues, remains a major translational limitation. Here, we provide an overview of oligonucleotide-based drug platforms, focusing on key approaches - including chemical modification, bioconjugation and the use of nanocarriers - which aim to address the delivery challenge.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Oligonucleotides
/
Gene Expression Regulation
/
Drug Delivery Systems
Limits:
Humans
Language:
En
Journal:
Nat Rev Drug Discov
Journal subject:
FARMACOLOGIA
/
TERAPIA POR MEDICAMENTOS
Year:
2020
Document type:
Article
Affiliation country:
United kingdom