Choi Andrew S, Moon Taylor J, Bhalotia Anubhuti, Rajan Aarthi, Ogunnaike Laolu, Hutchinson Diarmuid W, Hwang Inga, Gokhale Aaditya, Kim Justin N, Ma Timothy, Karathanasis Efstathios
Department of Biomedical Engineering, School of Medicine, Case Western Reserve University, Cleveland, Ohio 44106, United States.
Case Comprehensive Cancer Center, School of Medicine, Case Western Reserve University, Cleveland, Ohio 44106, United States.
Mol Pharm. 2025 Apr 7;22(4):1859-1868. doi: 10.1021/acs.molpharmaceut.4c01039. Epub 2025 Mar 4.
The engineerability of lipid nanoparticles (LNPs) and their ability to deliver nucleic acids make LNPs attractive tools for cancer immunotherapy. LNP-based gene delivery can be employed for various approaches in cancer immunotherapy, including encoding tumor-associated antigens and silencing of negative immune checkpoint proteins. For example, LNPs carrying small interfering RNAs can offer several advantages, including sustained and durable inhibition of an immune checkpoint protein. Due to their tunable design, modifying the lipid composition of LNPs can regulate the rate of their uptake by immune cells and the rate of gene silencing. Controlling the kinetics of LNP uptake provides additional flexibility and strategies to generate appropriate immunomodulation in the tumor microenvironment. Here, we evaluated the effects of polyethylene glycol (PEG) content ranging from 0.5 to 6 mol % on the cellular uptake of LNPs by immune cells and gene silencing of PD-L1 after intratumoral administration. We evaluated the cellular uptake and PD-L1 blockade in vitro in cell studies and in vivo using the YUMM1.7 melanoma tumor model. Cell studies showed that the rate of cell uptake was inversely correlated to an increasing mol % of PEG in a linear relationship. In the in vivo studies, 0.5% PEG LNP initiated an immediate effect in the tumor with a significant decrease in the PD-L1 expression of immune cells observed within 24 h. In comparison, the gene silencing effect of 6% PEG LNP was delayed, with a significant decrease of PD-L1 expression in immune cell subsets being observed 72 h after administration. Notably, performance of the 6% PEG LNP at 72 h was comparable to that of the 0.5% PEG LNP at 24 h. Overall, this study suggests that PEG modifications and intratumoral administration of LNPs can be a promising strategy for an effective antitumor immune response.
脂质纳米颗粒(LNPs)的可工程性及其递送核酸的能力,使LNPs成为癌症免疫治疗中具有吸引力的工具。基于LNP的基因递送可用于癌症免疫治疗的各种方法,包括编码肿瘤相关抗原和沉默负性免疫检查点蛋白。例如,携带小干扰RNA的LNPs可提供多种优势,包括对免疫检查点蛋白的持续抑制。由于其可调节的设计,改变LNPs的脂质组成可调节免疫细胞对其摄取的速率以及基因沉默的速率。控制LNP摄取的动力学为在肿瘤微环境中产生适当的免疫调节提供了额外的灵活性和策略。在此,我们评估了聚乙二醇(PEG)含量在0.5至6 mol%范围内对免疫细胞摄取LNPs以及瘤内给药后PD-L1基因沉默的影响。我们在细胞研究中以及使用YUMM1.7黑色素瘤肿瘤模型在体内评估了细胞摄取和PD-L1阻断情况。细胞研究表明,细胞摄取速率与PEG的mol%增加呈线性反比关系。在体内研究中,0.5% PEG的LNP在肿瘤中引发了即时效应,在24小时内观察到免疫细胞的PD-L1表达显著降低。相比之下,6% PEG的LNP的基因沉默效应延迟,在给药后72小时观察到免疫细胞亚群中的PD-L1表达显著降低。值得注意的是,6% PEG的LNP在72小时时的表现与0.5% PEG的LNP在24小时时相当。总体而言,这项研究表明,PEG修饰和LNPs的瘤内给药可能是一种有效的抗肿瘤免疫反应的有前景的策略。