相关实验视频

Updated: May 5, 2026

Protocol for MicroRNA Transfer into Adult Bone Marrow-derived Hematopoietic Stem Cells to Enable Cell Engineering Combined with Magnetic Targeting

Protocol for MicroRNA Transfer into Adult Bone Marrow-derived Hematopoietic Stem Cells to Enable Cell Engineering Combined with Magnetic Targeting

Published on: June 18, 2018

灵感来自于自然的平台纳米技术,用于将RNA传递给髓状细胞及其骨髓祖先

Stijn R J Hofstraat^1,2, Tom Anbergen³, Robby Zwolsman^1,2

¹Laboratory of Chemical Biology, Department of Biomedical Engineering, Eindhoven University of Technology, Eindhoven, the Netherlands.

Nature nanotechnology

|February 3, 2025

概括

一种新型的阿波利波蛋白纳米粒子 (aNP) 平台有效地将核酸疗法,包括小干扰RNA (siRNA),传递给骨髓细胞. 这一突破解决了系统稳定性和基因沉默和表达的有针对性传递方面的挑战.

科学领域:

生物技术是生物技术.
纳米医学是一种纳米医学.
基因治疗基因治疗

背景情况:

核酸治疗药物的系统传递和稳定性到骨髓细胞仍然是重大挑战.
针对性传递对于基因沉默,表达和编辑应用在体内至关重要.

研究的目的:

开发和描述一个纳米技术平台,使用天然脂蛋白进行核酸输送.
评估阿波利波蛋白纳米颗粒 (aNPs) 传递siRNA,反意义寡核酸和mRNA到骨髓髓髓细胞和血液造血干细胞和祖细胞的疗效.

主要方法:

开发包含siRNA的阿波利波蛋白纳米粒子 (aNP) 配方.
对aNP配方进行全面的物理化学表征和体外性能评估.
在体内评估aNP-siRNA用于在小鼠免疫细胞子集和瘤模型中的基因沉默.
证明aNP适用于反意义寡核酸拼接切换和基于mRNA的蛋白质生产.

主要成果:

一个aNP配方库被创建和描述.
选择的aNP-siRNA配方在体内证明了有效地抑制了溶解体相关的膜蛋白1 (Lamp1).
最有效的aNP配方在小鼠瘤模型中显示出治疗潜力.
通过交付核酸,aNP平台成功地促进了拼接切换和蛋白质生产.

结论:

阿波利波蛋白纳米粒子 (aNP) 平台展示了稳定和有针对性的多种核酸治疗药物的显著潜力.
这个平台解决了在达到骨髓寄居髓状细胞和造血干细胞和祖细胞的关键挑战.
aNP技术有望促进体内基因治疗和精准医学应用的进步.

更多相关视频

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

Polyethyleneimine-coated Iron Oxide Nanoparticles as a Vehicle for the Delivery of Small Interfering RNA to Macrophages In Vitro and In Vivo

Polyethyleneimine-coated Iron Oxide Nanoparticles as a Vehicle for the Delivery of Small Interfering RNA to Macrophages In Vitro and In Vivo

Published on: February 5, 2019

相关概念视频

Differentiation of Common Myeloid Progenitor Cells

Differentiation of Common Myeloid Progenitor Cells

Common myeloid progenitors (CMPs) are oligopotent cells that can differentiate into granulocytes and macrophages. Granulocytes and macrophages are essential for protecting the body against bacterial, viral, or fungal infections. They migrate from the bone marrow into the circulating blood to reach specific tissue sites where they differentiate and help in immune surveillance. However, they survive only for a few days and must be continuously made available to the organism to maintain a robust...