相关实验视频

Updated: May 26, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

系统地识别和表征高效率的Cas9指导RNA,用于ADAR的治疗向

Benjamin G Gowen¹, Kory Melton¹, Weng In Leong¹

¹Spotlight Therapeutics, Hayward, California, United States of America.

|February 24, 2025

概括

研究人员开发了CRISPR/Cas9指导RNA用于腺脱氨酶 (ADAR) 淘汰,诱导免疫反应. 这为基因组医学提供了一个新的途径,在癌症等疾病中准ADAR.

科学领域:

基因组医学是基因组医学.
分子生物学分子生物学
免疫学免疫学免疫学

背景情况:

氨酸脱氨酶 (ADAR) 是一种治疗点,在癌症和其他疾病中具有潜力.
对ADAR的有效和选择性抑制策略尚不清楚.
克里斯普尔/卡斯9技术提供了一种精确的基因编辑方法.

研究的目的:

通过使用CRISPR/Cas9.9来识别高效的Cas9指导RNA用于ADAR淘汰.
描述ADAR淘汰赛后的药理动力学和生物反应.
评估不同的ADAR异型在细胞反应中的作用.

主要方法:

针对ADAR向的多阶段导向RNA查.
在人类初级免疫细胞中指导RNA活性的表征.
评估剂量反应活性和目标外的特征.
免疫反应的分析,包括I型干扰素诱导.

主要成果:

确定了高效的Cas9指导RNA用于ADAR淘汰.
观察到的剂量反应活性和有利的计算非目标配置文件.
ADAR淘汰赛诱导了显著的免疫反应,包括I型干扰素.
针对ADAR的p150异型足以调解主要的生物反应.

结论:

为CRISPR/Cas9应用开发了一种具有良好的特征,高效的ADAR向导RNA的资源.
ADAR淘汰会引起免疫反应,突出其在dSRNA感知中的作用.
ADAR的p150异型似乎是观察到的生物效应的主要媒介.

更多相关视频

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Using Sniper-Cas9 to Minimize Off-target Effects of CRISPR-Cas9 Without the Loss of On-target Activity Via Directed Evolution

Using Sniper-Cas9 to Minimize Off-target Effects of CRISPR-Cas9 Without the Loss of On-target Activity Via Directed Evolution

Published on: February 26, 2019

相关概念视频

CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...

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...