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

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

doi:10.1016/j.omtn.2024.102224

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

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

Tel Aviv University

Research output: Contribution to journal › Review article › peer-review

5 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 language	English
Article number	102224
Journal	Molecular Therapy Nucleic Acids
Volume	35
Issue number	2
DOIs	https://doi.org/10.1016/j.omtn.2024.102224
State	Published - 11 Jun 2024
Externally published	Yes

Bibliographical note

Publisher Copyright:
© 2024 The Author(s)

Keywords

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

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10.1016/j.omtn.2024.102224

Cite this

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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.",

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AB - 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.

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

Abstract

Bibliographical note

Keywords

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