Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology , Rm 76-553, Cambridge, Massachusetts 02139, United States .
ACS Nano. 2013 Nov 26;7(11):9571-84. doi: 10.1021/nn4047925. Epub 2013 Oct 21.
A single nanoparticle platform has been developed through the modular and controlled layer-by-layer process to codeliver siRNA that knocks down a drug-resistance pathway in tumor cells and a chemotherapy drug to challenge a highly aggressive form of triple-negative breast cancer. Layer-by-layer films were formed on nanoparticles by alternately depositing siRNA and poly-l-arginine; a single bilayer on the nanoparticle surface could effectively load up to 3500 siRNA molecules, and the resulting LbL nanoparticles exhibit an extended serum half-life of 28 h. In animal models, one dose via intravenous administration significantly reduced the target gene expression in the tumors by almost 80%. By generating the siRNA-loaded film atop a doxorubicin-loaded liposome, we identified an effective combination therapy with siRNA targeting multidrug resistance protein 1, which significantly enhanced doxorubicin efficacy by 4 fold in vitro and led to up to an 8-fold decrease in tumor volume compared to the control treatments with no observed toxicity. The results indicate that the use of layer-by-layer films to modify a simple liposomal doxorubicin delivery construct with a synergistic siRNA can lead to significant tumor reduction in the cancers that are otherwise nonresponsive to treatment with Doxil or other common chemotherapy drugs. This approach provides a potential strategy to treat aggressive and resistant cancers, and a modular platform for a broad range of controlled multidrug therapies customizable to the cancer type in a singular nanoparticle delivery system.
已经开发出一种通过模块化和控制的层层工艺的单个纳米颗粒平台,用于共递 siRNA,以敲低肿瘤细胞中的耐药途径和化疗药物,以挑战三阴性乳腺癌的高度侵袭性形式。通过交替沉积 siRNA 和聚-l-精氨酸在纳米颗粒上形成层层膜;在纳米颗粒表面上的单个双层可以有效地装载多达 3500 个 siRNA 分子,并且所得的 LbL 纳米颗粒表现出延长的血清半衰期为 28 小时。在动物模型中,通过静脉内给药一次,可使肿瘤中的靶基因表达降低近 80%。通过在载多柔比星脂质体上生成负载 siRNA 的薄膜,我们确定了一种有效的联合治疗方法,该方法靶向多药耐药蛋白 1 的 siRNA,在体外显着提高了多柔比星的功效,与没有观察到毒性的对照治疗相比,肿瘤体积减少了 8 倍。结果表明,使用层层膜来修饰协同 siRNA 的简单脂质体多柔比星递送构建体可以导致对 Doxil 或其他常用化疗药物治疗无反应的癌症中的肿瘤显着减少。这种方法为治疗侵袭性和耐药性癌症提供了一种潜在的策略,并为在单一纳米颗粒递送系统中针对癌症类型定制的广泛的受控多药物治疗提供了一个模块化平台。
J Control Release. 2017-3-14
Adv Drug Deliv Rev. 2013-7-21
Biochem Biophys Res Commun. 2017-12-2
Exp Hematol Oncol. 2025-5-19
Drug Deliv Transl Res. 2025-4-23
Adv Mater. 2024-12
Adv Funct Mater. 2013-7-26
Angew Chem Int Ed Engl. 2013-6-17
Biomaterials. 2013-4-22