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Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing.
Nature Nanotechnology ( IF 38.3 ) Pub Date : 2020-04-06 , DOI: 10.1038/s41565-020-0669-6
Qiang Cheng 1 , Tuo Wei 1 , Lukas Farbiak 1 , Lindsay T Johnson 1 , Sean A Dilliard 1 , Daniel J Siegwart 1
Affiliation  

CRISPR-Cas gene editing and messenger RNA-based protein replacement therapy hold tremendous potential to effectively treat disease-causing mutations with diverse cellular origin. However, it is currently impossible to rationally design nanoparticles that selectively target specific tissues. Here, we report a strategy termed selective organ targeting (SORT) wherein multiple classes of lipid nanoparticles are systematically engineered to exclusively edit extrahepatic tissues via addition of a supplemental SORT molecule. Lung-, spleen- and liver-targeted SORT lipid nanoparticles were designed to selectively edit therapeutically relevant cell types including epithelial cells, endothelial cells, B cells, T cells and hepatocytes. SORT is compatible with multiple gene editing techniques, including mRNA, Cas9 mRNA/single guide RNA and Cas9 ribonucleoprotein complexes, and is envisioned to aid the development of protein replacement and gene correction therapeutics in targeted tissues.

中文翻译:

用于组织特异性 mRNA 递送和 CRISPR-Cas 基因编辑的选择性器官靶向 (SORT) 纳米颗粒。

CRISPR-Cas 基因编辑和基于信使 RNA 的蛋白质替代疗法在有效治疗具有多种细胞来源的致病突变方面具有巨大潜力。然而,目前不可能合理地设计选择性靶向特定组织的纳米颗粒。在这里,我们报告了一种称为选择性器官靶向 (SORT) 的策略,其中系统地设计了多种脂质纳米颗粒,通过添加补充的 SORT 分子来专门编辑肝外组织。靶向肺、脾和肝的 SORT 脂质纳米颗粒旨在选择性地编辑治疗相关的细胞类型,包括上皮细胞、内皮细胞、B 细胞、T 细胞和肝细胞。SORT 与多种基因编辑技术兼容,包括 mRNA、
更新日期:2020-04-06
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