Unlocking the Therapeutic Potential of Locked Nucleic Acids through Lipid Nanoparticle Delivery
Molecular Therapy - Nucleic Acids(2024)
摘要
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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关键词
Lipid Nanoparticles,Targeting,Antisense Oligonucleotides,Locked Nucleic Acids,Gene Delivery,Genetic Medicines
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