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增强的CRISPR-Cas9RNA系统传递使用细胞透基纳米颗粒用于高效的体外和体外应用

Veronica Guzman Gonzalez¹, Audrey Grunenberger², Olivier Nicoud³

¹University Grenoble Alpes, INSERM U 1209, CNRS UMR 5309, Cancer Targets and Experimental Therapeutics Team, Institute for Advanced Biosciences, 38000 Grenoble, France.; DivinCell SAS, Nimes, France.

Journal of controlled release : official journal of the Controlled Release Society

|November 9, 2024

概括

新型基于的纳米粒子有效地提供CRISPR-Cas9RNA用于基因编辑. 这些纳米粒子在体外和体内应用方面表现有前途,克服了遗传疾病治疗的交付挑战.

科学领域:

生物技术是生物技术.
基因治疗基因治疗
纳米医学是一种纳米医学.

背景情况:

CRISPR-Cas9基因编辑对癌症等遗传疾病具有治疗前景.
临床应用在提供CRISPR-Cas9组件,特别是RNA方面面临挑战,原因是降解和细胞吸收不良.
现有的传递方法与CRISPR-Cas9RNA的大小,电荷和水友性质相斗争.

研究的目的:

为了设计新的基于的纳米粒子 (ADGN),以有效地传递长RNACRISPR-Cas9系统.
评估ADGN纳米粒子用于基因编辑的体外和体内疗效和准能力.

主要方法:

设计了ADGN,用CRISPR-Cas9RNA形成自组装的纳米粒子.
评估了纳米粒子细胞膜穿越能力和通过拉米因受体向癌细胞.
在小鼠静脉注射后,在体外和体内评估了CRISPR-Cas9调解基因淘汰效率.

主要成果:

ADGN纳米颗粒有效地传递了CRISPR-Cas9RNA,在体外实现了60%的基因淘汰效率,首选G插入.
在小鼠中证明了成功的全身静脉注射,导致在正管肺瘤中有效的基因淘汰.
表明ADGN-RNA的体内分布受与RNA分子比率的影响.

结论:

基于ADGN的纳米颗粒代表了提供CRISPR-Cas9RNA系统的有希望的新平台.
这些纳米粒子克服了关键的传递障碍,使得在体外和体内都能有效编辑基因.
该研究强调了ADGN在开发针对遗传疾病和癌症的向基因疗法的潜力.

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