Department of Biological Sciences, KAIST Institute of BioCentury, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Daejeon, 34141, Republic of Korea.
Center for Precision Bio-Nanomedicine, KAIST, 291 Daehak-ro, Daejeon, 34141, Republic of Korea.
Adv Mater. 2024 Jul;36(27):e2311283. doi: 10.1002/adma.202311283. Epub 2024 Mar 21.
Organ-selective drug delivery is expected to maximize the efficacy of various therapeutic modalities while minimizing their systemic toxicity. Lipid nanoparticles and polymersomes can direct the organ-selective delivery of mRNAs or gene editing machineries, but their delivery is limited to mostly liver, spleen, and lung. A platform that enables delivery to these and other target organs is urgently needed. Here, a library of glycocalyx-mimicking nanoparticles (GlyNPs) comprising five randomly combined sugar moieties is generated, and direct in vivo library screening is used to identify GlyNPs with preferential biodistribution in liver, spleen, lung, kidneys, heart, and brain. Each organ-targeting GlyNP hit show cellular tropism within the organ. Liver, kidney, and spleen-targeting GlyNP hits equipped with therapeutics effectively can alleviate the symptoms of acetaminophen-induced liver injury, cisplatin-induced kidney injury, and immune thrombocytopenia in mice, respectively. Furthermore, the differential organ targeting of GlyNP hits is influenced not by the protein corona but by the sugar moieties displayed on their surface. It is envisioned that the GlyNP-based platform may enable the organ- and cell-targeted delivery of therapeutic cargoes.
器官选择性药物递送有望最大限度地提高各种治疗模式的疗效,同时将其全身毒性降至最低。脂质纳米粒和聚合物囊泡可以指导 mRNA 或基因编辑机器的器官选择性递送,但它们的递送仅限于肝脏、脾脏和肺部。迫切需要一种能够将药物递送到这些和其他靶器官的平台。在这里,生成了由五个随机组合的糖基组成的糖萼模拟纳米颗粒 (GlyNP) 文库,并直接进行体内文库筛选,以鉴定在肝脏、脾脏、肺、肾脏、心脏和大脑中具有优先分布的 GlyNP。每种靶向器官的 GlyNP 都显示出在器官内的细胞趋向性。携带治疗药物的肝、肾和脾靶向 GlyNP 有效缓解了对乙酰氨基酚引起的肝损伤、顺铂引起的肾损伤和免疫性血小板减少症在小鼠中的症状。此外,GlyNP 命中的差异器官靶向性不受蛋白质冠的影响,而是受其表面显示的糖基的影响。可以预见,基于 GlyNP 的平台可能能够实现治疗性货物的器官和细胞靶向递送。