A breath of fresh air: targeted non-viral in vivo gene correction in the mammalian lung

IF 40.8 1区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Signal Transduction and Targeted Therapy Pub Date : 2024-10-14 DOI:10.1038/s41392-024-01994-y
Jixin Liu, Dirk Grimm
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Abstract

In a recent study published in Science,1 Sun and colleagues showcase the power and potential of lung SORT LNPs, i.e., lipid nanoparticles that upon systemic delivery in mice specifically and efficiently target cells in the lung, most likely facilitated by their binding to plasma vitronectin and uptake via the vitronectin receptor. Most remarkably, when engineered to deliver a base editor, peripheral injection of SORT LNPs enabled highly efficient gene correction in lung stem cells, whole lung and trachea in a mouse model of cystic fibrosis, illustrating the enormous promise of this novel technology for human patients suffering from this devastating disease (Fig. 1).

Fig. 1
Abstract Image

Lipid nanoparticles (LNPs) bind to vitronectin, which facilitates their uptake by vitronectin receptors (VtnR) in the lungs. The figure illustrates the efficiency of gene editing in various lung cell types and the restoration of CFTR function. This figure was created with BioRender

Full size image

Abstract Image

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新鲜空气:哺乳动物肺部的定向非病毒体内基因校正
在最近发表于《科学》(Science)1 的一项研究中,Sun 及其同事展示了肺部 SORT LNPs 的威力和潜力,即脂质纳米粒子在小鼠体内全身注射后可特异性地高效靶向肺部细胞,这很可能是由于它们与血浆玻璃连蛋白结合并通过玻璃连蛋白受体被吸收。最引人注目的是,当设计用于递送碱基编辑器时,外周注射 SORT LNPs 能在囊性纤维化小鼠模型的肺干细胞、全肺和气管中实现高效的基因校正,这说明了这种新型技术在人类这种毁灭性疾病患者身上的巨大前景(图 1)。该图说明了基因编辑在各种肺细胞类型中的效率以及 CFTR 功能的恢复情况。本图使用 BioRenderFull size 图像创建
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来源期刊
Signal Transduction and Targeted Therapy
Signal Transduction and Targeted Therapy Biochemistry, Genetics and Molecular Biology-Genetics
CiteScore
44.50
自引率
1.50%
发文量
384
审稿时长
5 weeks
期刊介绍: Signal Transduction and Targeted Therapy is an open access journal that focuses on timely publication of cutting-edge discoveries and advancements in basic science and clinical research related to signal transduction and targeted therapy. Scope: The journal covers research on major human diseases, including, but not limited to: Cancer,Cardiovascular diseases,Autoimmune diseases,Nervous system diseases.
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