Abstract
Locked nucleic acids (LNAs) are a subtype of antisense oligonucleotides (ASOs) that are characterized by a bridge within the sugar moiety. LNAs owe their robustness to this chemical modification, which as the name suggests, locks it in one conformation. This perspective includes two components: a general overview on ASOs from one side and on delivery issues focusing on lipid nanoparticles (LNPs) on the other side. Throughout, a screening of the ongoing clinical trials involving ASOs is given, as well as a take on the versatility and challenges of using LNAs. Finally, we highlight the potential of LNPs as carriers for the successful delivery of LNAs.
Original language | English (US) |
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Article number | 102224 |
Journal | Molecular Therapy - Nucleic Acids |
Volume | 35 |
Issue number | 2 |
DOIs | |
State | Published - Jun 11 2024 |
Keywords
- MT: Oligonucleotides: Therapies and Applications
- antisense oligonucleotides
- gene delivery
- genetic medicine
- lipid nanoparticles
- locked nucleic acids
- targeting
ASJC Scopus subject areas
- Molecular Medicine
- Drug Discovery