{"title":"新鲜空气：哺乳动物肺部的定向非病毒体内基因校正","authors":"Jixin Liu, Dirk Grimm","doi":"10.1038/s41392-024-01994-y","DOIUrl":null,"url":null,"abstract":"<p>In a recent study published in <i>Science</i>,<sup>1</sup> Sun and colleagues showcase the power and potential of lung SORT LNPs, <i>i.e</i>., 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).</p><figure><figcaption><b data-test=\"figure-caption-text\">Fig. 1</b></figcaption><picture><source srcset=\"//media.springernature.com/lw685/springer-static/image/art%3A10.1038%2Fs41392-024-01994-y/MediaObjects/41392_2024_1994_Fig1_HTML.png?as=webp\" type=\"image/webp\"/><img alt=\"figure 1\" aria-describedby=\"Fig1\" height=\"144\" loading=\"lazy\" src=\"//media.springernature.com/lw685/springer-static/image/art%3A10.1038%2Fs41392-024-01994-y/MediaObjects/41392_2024_1994_Fig1_HTML.png\" width=\"685\"/></picture><p>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</p><span>Full size image</span><svg aria-hidden=\"true\" focusable=\"false\" height=\"16\" role=\"img\" width=\"16\"><use xlink:href=\"#icon-eds-i-chevron-right-small\" xmlns:xlink=\"http://www.w3.org/1999/xlink\"></use></svg></figure>","PeriodicalId":40,"journal":{"name":"Inorganic Chemistry","volume":"28 1","pages":""},"PeriodicalIF":4.3000,"publicationDate":"2024-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"A breath of fresh air: targeted non-viral in vivo gene correction in the mammalian lung\",\"authors\":\"Jixin Liu, Dirk Grimm\",\"doi\":\"10.1038/s41392-024-01994-y\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>In a recent study published in <i>Science</i>,<sup>1</sup> Sun and colleagues showcase the power and potential of lung SORT LNPs, <i>i.e</i>., 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).</p><figure><figcaption><b data-test=\\\"figure-caption-text\\\">Fig. 1</b></figcaption><picture><source srcset=\\\"//media.springernature.com/lw685/springer-static/image/art%3A10.1038%2Fs41392-024-01994-y/MediaObjects/41392_2024_1994_Fig1_HTML.png?as=webp\\\" type=\\\"image/webp\\\"/><img alt=\\\"figure 1\\\" aria-describedby=\\\"Fig1\\\" height=\\\"144\\\" loading=\\\"lazy\\\" src=\\\"//media.springernature.com/lw685/springer-static/image/art%3A10.1038%2Fs41392-024-01994-y/MediaObjects/41392_2024_1994_Fig1_HTML.png\\\" width=\\\"685\\\"/></picture><p>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. 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A breath of fresh air: targeted non-viral in vivo gene correction in the mammalian lung
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
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
期刊介绍：
Inorganic Chemistry publishes fundamental studies in all phases of inorganic chemistry. Coverage includes experimental and theoretical reports on quantitative studies of structure and thermodynamics, kinetics, mechanisms of inorganic reactions, bioinorganic chemistry, and relevant aspects of organometallic chemistry, solid-state phenomena, and chemical bonding theory. Emphasis is placed on the synthesis, structure, thermodynamics, reactivity, spectroscopy, and bonding properties of significant new and known compounds.
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