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通过脂质纳米颗粒递送释放锁核酸的治疗潜力。

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

作者信息

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

机构信息

Laboratory of Precision NanoMedicine, Shmunis School for Biomedicine and Cancer Research, George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel Aviv 69978, Israel.

Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv 69978, Israel.

出版信息

Mol Ther Nucleic Acids. 2024 May 20;35(2):102224. doi: 10.1016/j.omtn.2024.102224. eCollection 2024 Jun 11.

DOI:10.1016/j.omtn.2024.102224
PMID:38933259
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11201112/
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.

摘要

锁核酸（LNAs）是反义寡核苷酸（ASOs）的一种亚型，其特征在于糖部分内有一个桥连结构。LNAs的稳定性归功于这种化学修饰，顾名思义，它将核酸锁定在一种构象中。这一观点包括两个方面：一方面是对ASOs的总体概述，另一方面是聚焦于脂质纳米颗粒（LNPs）的递送问题。文中还给出了对正在进行的涉及ASOs的临床试验的筛选，以及对使用LNAs的多功能性和挑战的看法。最后，我们强调了LNPs作为成功递送LNAs载体的潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/1e88bbe1eeff/gr6.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/e60cecd90ccc/fx1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/667eb78ebceb/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/8884bad0133c/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/0c47c5b8ee44/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/ce22062e095f/gr4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/6e72821f22bb/gr5.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/1e88bbe1eeff/gr6.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/e60cecd90ccc/fx1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/667eb78ebceb/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/8884bad0133c/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/0c47c5b8ee44/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/ce22062e095f/gr4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/6e72821f22bb/gr5.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/13a0/11201112/1e88bbe1eeff/gr6.jpg

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