抗 SARS-CoV-2 非结构蛋白 Nsp9 的纳米抗体可抑制病毒在人气道上皮细胞中的复制

IF 6.5 2区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Molecular Therapy. Nucleic Acids Pub Date : 2024-08-15 DOI:10.1016/j.omtn.2024.102304
Tomas Venit, Jeremy Blavier, Sibusiso B. Maseko, Sam Shu, Lilia Espada, Christopher Breunig, Hans-Peter Holthoff, Sabrina C. Desbordes, Martin Lohse, Gennaro Esposito, Jean-Claude Twizere, Piergiorgio Percipalle
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引用次数: 0

摘要

纳米抗体正在成为药物设计的重要工具。最近有几种纳米抗体通过靶向表面暴露的尖峰蛋白,成为严重急性呼吸系统综合症冠状病毒(SARS-CoV-2)进入宿主细胞的抑制剂。在这里,我们建立了一个管道,它的目标是高度保守的病毒蛋白,这些蛋白只有在病毒进入宿主细胞后,当基于 SARS-CoV-2 RNA 的基因组被翻译时才会产生。作为原理验证,我们设计了抗SARS-CoV-2非结构蛋白(Nsp)9的纳米抗体,病毒基因组复制需要这种蛋白。其中一种抗 Nsp9 纳米抗体 2NSP23 以前曾利用免疫测定和核磁共振光谱绘制表位图对其进行过表征,我们表达了这种纳米抗体并发现它能特异性地阻断 SARS-CoV-2 的复制。接下来,我们将 2NSP23 纳米抗体作为 mRNA 封装到脂质纳米粒子(LNPs)中。通过 qPCR 和 RNA 深度测序,我们发现这种纳米抗体(在此称为 LNP-mRNA-2NSP23)能在细胞内被内化和翻译,并能抑制多种 SARS-CoV-2 变体。这些结果在模拟肺组织外表面的气液界面三维重建人体上皮细胞中得到了证实。这些观察结果表明,LNP-mRNA-2NSP23 被内化,翻译后通过靶向活细胞中的 Nsp9 抑制病毒复制。我们推测,LNP-mRNA-2NSP23 有可能被转化为一种创新策略,产生新型抗病毒药物,对冠状病毒非常有效。
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Nanobody against SARS-CoV-2 non-structural protein Nsp9 inhibits viral replication in human airway epithelia
Nanobodies are emerging as critical tools for drug design. Several have been recently created to serve as inhibitors of severe acute respiratory syndrome coronavirus s (SARS-CoV-2) entry in the host cell by targeting surface-exposed spike protein. Here we have established a pipeline that instead targets highly conserved viral proteins made only after viral entry into the host cell when the SARS-CoV-2 RNA-based genome is translated. As proof of principle, we designed nanobodies against the SARS-CoV-2 non-structural protein (Nsp)9, which is required for viral genome replication. One of these anti-Nsp9 nanobodies, 2NSP23, previously characterized using immunoassays and nuclear magnetic resonance spectroscopy for epitope mapping, was expressed and found to block SARS-CoV-2 replication specifically. We next encapsulated 2NSP23 nanobody into lipid nanoparticles (LNPs) as mRNA. We show that this nanobody, hereby referred to as LNP-mRNA-2NSP23, is internalized and translated in cells and suppresses multiple SARS-CoV-2 variants, as seen by qPCR and RNA deep sequencing. These results are corroborated in three-dimensional reconstituted human epithelium kept at air-liquid interface to mimic the outer surface of lung tissue. These observations indicate that LNP-mRNA-2NSP23 is internalized and, after translation, it inhibits viral replication by targeting Nsp9 in living cells. We speculate that LNP-mRNA-2NSP23 may be translated into an innovative strategy to generate novel antiviral drugs highly efficient across coronaviruses.
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来源期刊
Molecular Therapy. Nucleic Acids
Molecular Therapy. Nucleic Acids MEDICINE, RESEARCH & EXPERIMENTAL-
CiteScore
15.40
自引率
1.10%
发文量
336
审稿时长
20 weeks
期刊介绍: Molecular Therapy Nucleic Acids is an international, open-access journal that publishes high-quality research in nucleic-acid-based therapeutics to treat and correct genetic and acquired diseases. It is the official journal of the American Society of Gene & Cell Therapy and is built upon the success of Molecular Therapy. The journal focuses on gene- and oligonucleotide-based therapies and publishes peer-reviewed research, reviews, and commentaries. Its impact factor for 2022 is 8.8. The subject areas covered include the development of therapeutics based on nucleic acids and their derivatives, vector development for RNA-based therapeutics delivery, utilization of gene-modifying agents like Zn finger nucleases and triplex-forming oligonucleotides, pre-clinical target validation, safety and efficacy studies, and clinical trials.
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