Australian Centre for Nanomedicine and Centre for Advanced Macromolecular Design, School of Chemical Engineering, University of New South Wales, Sydney, 2052, Australia.
Storr Liver Unit, Westmead Millenium Institute and Westmead Hospital, University of Sydney, Sydney, NSW, Australia.
Small. 2015 May 20;11(19):2291-304. doi: 10.1002/smll.201402870. Epub 2015 Jan 13.
Polymeric nanoparticles are designed to transport and deliver nitric oxide (NO) into hepatic stellate cells (HSCs) for the potential treatment of both liver fibrosis and portal hypertension. The nanoparticles, incorporating NO donor molecules (S-nitrosoglutathione compound), are designed for liver delivery, minimizing systemic delivery of NO. The nanoparticles are decorated with vitamin A to specifically target HSCs. We demonstrate, using in vitro and in vivo experiments, that the targeted nanoparticles are taken up specifically by rat primary HSCs and the human HSC cell line accumulating in the liver. When nanoparticles, coated with vitamin A, release NO in liver cells, we find inhibition of collagen I and α-smooth muscle actin (α-SMA), fibrogenic genes associated with activated HSCs expression in primary rat liver and human activated HSCs without any obvious cytotoxic effects. Finally, NO-releasing nanoparticles targeted with vitamin A not only attenuate endothelin-1 (ET-1) which elicites HSC contraction but also acutely alleviates haemodynamic disorders in bile duct-ligated-induced portal hypertension evidenced by decreasing portal pressure (≈20%) and unchanging mean arterial pressure. This study clearly shows, for the first time, the potential for HSC targeted nanoparticle delivery of NO as a treatment for liver diseases with proven efficacy for alleviating both liver fibrosis and portal hypertension.
聚合物纳米粒子被设计用于向肝星状细胞(HSCs)输送和递送一氧化氮(NO),以潜在治疗肝纤维化和门静脉高压症。这些纳米粒子包含一氧化氮供体分子(S-亚硝基谷胱甘肽化合物),旨在用于肝脏递药,最大限度地减少 NO 的全身递送。纳米粒子用维生素 A 进行修饰,以特异性靶向 HSCs。我们通过体外和体内实验证明,靶向纳米粒子被大鼠原代 HSCs 和人 HSC 细胞系特异性摄取,并在肝脏中积累。当涂覆有维生素 A 的纳米粒子在肝细胞中释放 NO 时,我们发现其抑制胶原 I 和 α-平滑肌肌动蛋白(α-SMA)的表达,这些是与活化的 HSCs 表达相关的纤维生成基因,而对原代大鼠肝和人活化的 HSCs 没有明显的细胞毒性作用。最后,用维生素 A 靶向的 NO 释放纳米粒子不仅可以减轻内皮素-1(ET-1)引起的 HSC 收缩,而且还可以通过降低门静脉压(≈20%)和不变的平均动脉压来急性缓解胆管结扎诱导的门静脉高压症的血流动力学紊乱。这项研究首次清楚地表明,将 NO 靶向 HSC 的纳米粒子递药作为治疗肝脏疾病的一种方法具有很大的潜力,已被证明可有效缓解肝纤维化和门静脉高压症。