可吸入的完碳RNA纳米囊克服了治疗肺转移的生物障碍
Kasturi Siddhanta1, Neha Kumari1, Aditya Sundar2
1Department of Pharmaceutical Sciences, College of Pharmacy, University of Nebraska Medical Center, Omaha, NE 68198, USA; Center for Drug Delivery and Nanomedicine, University of Nebraska Medical Center, Omaha, NE 68198, USA.
概括
新的纳米囊有效地向肺部提供RNA疗法,克服生物障碍. 肺部疾病治疗的这一突破显示出改善患者治疗结果的希望.
科学领域:
- 生物医学工程 生物医学工程
- 纳米技术 纳米技术
- 肺部医学 肺部医学
背景情况:
- 吸入RNA疗法面临着粘液捕获和免疫清除等挑战.
- 有效地将RNA输送到目标肺细胞仍然是治疗肺部疾病的重要障碍.
研究的目的:
- 开发基于稳定气溶 perfluorocarbon (PFC) 的miRNA纳米囊,以增强向肺部输送RNA.
- 克服阻止肺部疾病有效RNA治疗的生物障碍.
主要方法:
- 开发了基于PFC的miRNA纳米囊,由PAMD-C稳定,一种胆固醇修饰的聚合物模拟.
- 评估纳米囊免疫反应,粘液运输,细胞吸收和肺部保留在一个正型肺转移模型.
- 评估了miR-34a模仿装载纳米囊在抑制转移性外生和改善生存的治疗效果.
主要成果:
- 这些纳米囊表现出微不足道的免疫清除,最小的炎症反应和高效的粘液传输.
- 超过60%的癌细胞和大多数肺上皮细胞在一次剂量后内化了纳米囊,其保留时间超过48小时.
- 用miR-34a模拟载荷纳米囊治疗使中位生存时间翻了一番,并抑制了转移性增长.
结论:
- 开发的基于PFC的纳米囊有效地克服了可吸入RNA药物的生物障碍.
- 这个平台提供了一个可翻译的方法来治疗各种肺部疾病,包括肺癌.
- 纳米囊允许重复剂量,因为可以忽略的细胞因子释放和最小的炎症反应.
相关概念视频
01:23RNA Interference
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
02:15Experimental RNAi
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...