Center for Nanotechnology in Drug Delivery and Division of Pharmacoengineering and Molecular Therapeutics, Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, North Carolina 27599, United States.
Department of Physics, Howell Science Complex, East Carolina University, Greenville, North Carolina 27858, United States.
ACS Appl Mater Interfaces. 2023 Apr 26;15(16):19877-19891. doi: 10.1021/acsami.3c00179. Epub 2023 Apr 11.
Engineered cells used as smart vehicles for delivery of secreted therapeutic proteins enable effective treatment of cancer and certain degenerative, autoimmune, and genetic disorders. However, current cell-based therapies use mostly invasive tools for tracking proteins and do not allow for controlled secretion of therapeutic proteins, which could result in unconstrained killing of surrounding healthy tissues or ineffective killing of host cancer cells. Regulating the expression of therapeutic proteins after success of therapy remains elusive. In this study, a noninvasive therapeutic approach mediated by magneto-mechanical actuation (MMA) was developed to remotely regulate the expression of the tumor necrosis factor-related apoptosis-inducing ligand (TRAIL) protein, which is secreted by transduced cells. Stem cells, macrophages, and breast cancer cells were transduced with a lentiviral vector encoding the SGpL2TR protein. SGpL2TR comprises TRAIL and GpLuc domains optimized for cell-based applications. Our approach relies on the remote actuation of cubic-shape highly magnetic field responsive superparamagnetic iron oxide nanoparticles (SPIONs) coated with nitrodopamine PEG (ND-PEG), which are internalized within the cells. Cubic ND-PEG-SPIONs actuated by superlow frequency alternating current magnetic fields can translate magnetic forces into mechanical motion and in turn spur mechanosensitive cellular responses. Cubic ND-PEG-SPIONs were artificially designed to effectively operate at low magnetic field strengths (<100 mT) retaining approximately 60% of their saturation magnetization. Compared to other cells, stems cells were more sensitive to the interaction with actuated cubic ND-PEG-SPIONs, which clustered near the endoplasmic reticulum (ER). Luciferase, ELISA, and RT-qPCR analyses revealed a marked TRAIL downregulation (secretion levels were depleted down to 30%) when intracellular particles at 0.100 mg/mL Fe were actuated by magnetic fields (65 mT and 50 Hz for 30 min). Western blot studies indicated actuated, intracellular cubic ND-PEG-SPIONs can cause mild ER stress at short periods (up to 3 h) of postmagnetic field treatment thus leading to the unfolded protein response. We observed that the interaction of TRAIL polypeptides with ND-PEG can also contribute to this response. To prove the applicability of our approach, we used glioblastoma cells, which were exposed to TRAIL secreted from stem cells. We demonstrated that in the absence of MMA treatment, TRAIL essentially killed glioblastoma cells indiscriminately, but when treated with MMA, we were able to control the cell killing rate by adjusting the magnetic doses. This approach can expand the capabilities of stem cells to serve as smart vehicles for delivery of therapeutic proteins in a controlled manner without using interfering and expensive drugs, while retaining their potential to regenerate damaged tissue after treatment. This approach brings forth new alternatives to regulate protein expression noninvasively for cell therapy and other cancer therapies.
作为传递分泌型治疗蛋白的智能载体的工程细胞可有效治疗癌症和某些退行性、自身免疫性和遗传性疾病。然而,目前的基于细胞的疗法主要使用侵入性工具来跟踪蛋白,并且不允许治疗蛋白的受控分泌,这可能导致对周围健康组织的不受控制的杀伤或宿主癌细胞的无效杀伤。在治疗成功后调节治疗蛋白的表达仍然难以捉摸。在这项研究中,开发了一种由磁机械致动(MMA)介导的非侵入性治疗方法,以远程调节转导细胞分泌的肿瘤坏死因子相关凋亡诱导配体(TRAIL)蛋白的表达。干细胞、巨噬细胞和乳腺癌细胞被慢病毒载体转导,该载体编码 SGpL2TR 蛋白。SGpL2TR 包含 TRAIL 和 GpLuc 结构域,针对细胞应用进行了优化。我们的方法依赖于远程激活涂有硝丁基多巴胺 PEG(ND-PEG)的立方形状高磁场响应超顺磁性氧化铁纳米粒子(SPION),这些纳米粒子被细胞内化。在超低频交流磁场作用下,立方 ND-PEG-SPION 可以将磁力转化为机械运动,并反过来刺激机械敏感的细胞反应。立方 ND-PEG-SPION 被人为设计为在低磁场强度(<100 mT)下有效运行,保留其约 60%的饱和磁化强度。与其他细胞相比,干细胞对与被激活的立方 ND-PEG-SPION 的相互作用更敏感,这些 SPION 聚集在内质网(ER)附近。荧光素酶、ELISA 和 RT-qPCR 分析显示,当以 0.100mg/mL Fe 的浓度向细胞内颗粒施加磁场(65mT 和 50Hz 持续 30min)时,TRAIL 的表达明显下调(分泌水平降低至 30%)。Western blot 研究表明,在磁场处理后短时间内(长达 3 小时),被激活的细胞内立方 ND-PEG-SPION 会引起轻度内质网应激,从而导致未折叠蛋白反应。我们观察到 TRAIL 多肽与 ND-PEG 的相互作用也可能导致这种反应。为了证明我们方法的适用性,我们使用胶质母细胞瘤细胞,这些细胞暴露于干细胞分泌的 TRAIL 下。我们证明,在没有 MMA 治疗的情况下,TRAIL 基本上不加区别地杀死胶质母细胞瘤细胞,但在用 MMA 治疗时,我们能够通过调整磁剂量来控制细胞杀伤率。这种方法可以扩展干细胞作为治疗蛋白的智能载体的功能,以可控的方式进行传递,而无需使用干扰和昂贵的药物,同时保留它们在治疗后再生受损组织的潜力。这种方法为细胞治疗和其他癌症治疗带来了新的选择,可以非侵入性地调节蛋白表达。