相关实验视频

Updated: Jun 22, 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

通过AAV提供CRISPR/Cas9系统作为基因治疗的载体

Yanan Wang¹, Haibin Jiang², Mopu Li²

¹Department of Neonatology, The Second School of Medicine, The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University, Wenzhou, Zhejiang, China; Department of Anesthesiology, 1st Affiliated Hospital of Wenzhou Medical University, Wenzhou, Zhejiang, China; The Second School of Medicine, Wenzhou Medical University, Wenzhou, Zhejiang, China.

|June 30, 2024

概括

腺相关病毒 (AAV) 提供CRISPR/Cas9基因编辑用于遗传疾病. 挑战包括AAV限制和Cas9免疫性,但正在开发有效基因治疗的对策.

科学领域:

分子生物学分子生物学
基因治疗基因治疗
病毒学病毒学

背景情况:

基因相关病毒 (AAV) 是一种高效和特定的基因疗法载体.
CRISPR/Cas9是一种强大的基因编辑系统,利用Cas9核酶和引导RNA.
结合AAV和CRISPR/Cas9显示出治疗遗传疾病和病毒感染的前景.

研究的目的:

审查AAV-CRISPR/Cas9系统在基因治疗中的作用.
确定与使用AAV和CRISPR/Cas9相关的挑战.
讨论应对这些挑战的潜在对策.

主要方法:

审查最近的动物实验和临床研究.
对AAV矢量特性和CRISPR/Cas9系统功能的分析.
确定临床挑战和提出的解决方案.

主要成果:

在临床前和临床环境中,AAV-CRISPR/Cas9系统显示出显著的潜力.
关键的挑战包括AAV免疫性,毒性和输送限制 (大脑,耳朵).
Cas9蛋白还带来了与免疫性和非标效应相关的挑战.

结论:

AAV-CRISPR/Cas9系统是基因治疗的一个有前途的工具.
克服载体传递,免疫性和特异性的挑战对于临床翻译至关重要.
目前正在进行的研究重点是开发有效的对策,以提高安全性和有效性.

关键词:

在 AAV AAV AAV 中.这就是CRISPR/Cas9的作用.基因治疗是一种基因疗法.

更多相关视频

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection

Published on: May 23, 2016

相关概念视频

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

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...