相关实验视频

Updated: Jan 17, 2026

Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells

Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells

Published on: June 10, 2022

开发BMX siRNA脂质纳米颗粒使用通过人工智能和癌症治疗实验发现的新型电离和可切割脂质

Abdulaziz Alasmari¹, Rwan Elsanhoury¹, Avinash Kumar¹

¹Division of Pharmaceutical Sciences, Arnold and Marie Schwartz College of Pharmacy and Health Sciences, Long Island University, Brooklyn, NY, USA.

International journal of pharmaceutics

|September 17, 2025

概括

新型维生素E衍生的脂质被用于制造脂质纳米粒子 (LNP),用于向癌症中的X染色体 (BMX) 上的骨髓. 这些BMX siRNA LNP在体外和体内表现出显著的抗癌活性,提供了一个有前途的新疗法方法.

科学领域:

纳米医学和药物输送
癌症治疗方法癌症治疗方法
分子生物学分子生物学

背景情况:

X染色体上的骨髓 (BMX) 是先进癌症治疗的新兴目标.
之前的研究利用了小分子模式来准BMX.
这项研究扩展了准策略,包括BMX siRNA脂质纳米粒子 (LNP).

研究的目的:

来自维生素E的设计,合成和表征新的可电离和可切割脂质.
为增强癌症治疗开发和优化BMX siRNA LNP.
评估这些新型LNP的体外和体内疗效.

主要方法:

脂质是从维生素E的γ-T3异构体中合成的,并使用1H NMR进行了表征.
人工智能 (AI) 和设计质量 (QbD) 被用于最佳的LNP配方.
使用甲醇稀释和挤出制备LNP,然后以物理化学性质和体外/体外抗癌活性进行表征.

主要成果:

优化的BMX siRNA LNPs的粒子大小为151nm,泽塔电位为30mV,具有pH依赖的释放.
试验室研究显示,对DU145和PC3前列腺癌细胞系具有显著的抗癌活性 (IC50值分别为1.1μM和1.3μM).
BMX siRNA LNPs显著降低了BMX mRNA的表达,并在动物模型中表现出强大的体内抗癌活性.

结论:

新型基于维生素E的可电离和可切割脂质使得有效的BMX siRNA LNP的开发成为可能.
优化的LNP显示了前列腺癌和潜在的其他BMX相关恶性瘤的有前途的治疗潜力.
这项工作支持基于LNP的治疗方法的进步,针对特定的癌症途径.

关键词:

BMX和新的目标目标癌症疗法开发开发癌症疗法开发发现制药公司的药品.配方开发配方开发脂质纳米颗粒是一种纳米粒子.新型的电离性脂质新型的电离性脂质在QbD和AI方面.翻译性药物学翻译性药物学

更多相关视频

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Published on: January 20, 2023

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

相关概念视频

siRNA - Small Interfering RNAs

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...

Experimental RNAi

Experimental 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...