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

Shahd Qassem, Dor Breier, Gonna Somu Naidu, Inbal Hazan-Halevy, Dan Peer

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

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 languageEnglish (US)
Article number102224
JournalMolecular Therapy - Nucleic Acids
Volume35
Issue number2
DOIs
StatePublished - 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

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