相关实验视频

Updated: Jun 23, 2026

Using Sniper-Cas9 to Minimize Off-target Effects of CRISPR-Cas9 Without the Loss of On-target Activity Via Directed Evolution
11:37

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

9.6K

聚焦超声波广泛扩大了AAV提供的Cas9分布和活动

Emrah Gumusgoz1, Sahba Kasiri1, Ibrahim Youssef2,3,4

  • 1Division of Neurology, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, 75390, USA.

Gene therapy
|February 1, 2025
PubMed
概括

聚焦超声波 (FUS) 通过暂时打开血脑屏障 (BBB) 来显著增强通过腺相关病毒 (AAV) 向大脑的基因传递. 这种方法成功地减少了Lafora病的小鼠模型中的疾病病理,为治疗遗传神经系统疾病提供了新的希望.

更多相关视频

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization08:20

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

Published on: September 2, 2021

4.1K
Focused Ultrasound Induced Blood-Brain Barrier Opening for Targeting Brain Structures and Evaluating Chemogenetic Neuromodulation08:37

Focused Ultrasound Induced Blood-Brain Barrier Opening for Targeting Brain Structures and Evaluating Chemogenetic Neuromodulation

Published on: December 22, 2020

3.5K

相关概念视频

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...