Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery

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.

Keywords

• MT: Oligonucleotides: Therapies and Applications
• lipid nanoparticles
• antisense oligonucleotides
• locked nucleic acids
• gene delivery
• genetic medicine