相关实验视频

Updated: Jan 6, 2026

CIRCLE-Seq for Interrogation of Off-Target Gene Editing

CIRCLE-Seq for Interrogation of Off-Target Gene Editing

Published on: November 1, 2024

保护基因组:在CRISPR/Cas9系统中用于目标外检测和最小化策略

Malihe Lotfi^1,2, Zahra Farshchian Yazdi³, Mohammad Reza Hashemi⁴

¹Medical Genetics Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.

Expert review of molecular diagnostics

|November 16, 2025

概括

克里斯普尔基因组编辑提供了精确的基因改造,但面临着非目标效应的挑战. 本综述探讨了用于更安全的临床应用,检测和最小化这些意外突变的策略.

科学领域:

分子生物学分子生物学
遗传学遗传学是一种遗传学.
生物技术是生物技术.

背景情况:

克里斯普尔-卡斯基因组编辑使精确的基因修改成为可能,彻底改变了诊断和治疗.
非目标突变是一个重大问题,可能导致基因组不稳定,并影响临床安全.
确保CRISPR技术的安全性和有效性需要解决非目标效应.

研究的目的:

在CRISPR基因组编辑中全面审查检测和减轻非目标效应的策略.
分析各种检测方法,包括in silico,in vitro和in vivo方法.
检查旨在提高基因组编辑精度和安全性的进展.

主要方法:

在体内,体外和体内方法用于检测非目标效应.
对指导RNA (gRNA) 工程和改进的Cas变体的分析.
优化传递系统,基质/主要编辑和抗CRISPR蛋白质的评估.

主要成果:

为了检测CRISPR的目标外效应,存在各种各样的方法,每个方法都有特定的优势和局限性.
目前正在开发各种策略,包括gRNA优化,新型Cas变体和先进的编辑技术,以提高特异性.
在缓解无意的基因组修改方面取得了进展.

结论:

对意外基因组修饰的精确体内评估和长期安全性仍然是关键的挑战.
未来的研究应该优先考虑高保真CRISPR变体,精细的计算模型和集成检测系统.
交付机制和监管框架的进步对于CRISPR疗法的临床转化至关重要.

关键词:

克里斯普尔是什么意思?克里斯普尔是什么意思?基因治疗的基因疗法基因组编辑基因组编辑非目标效应的非目标效应精准医学是一门精准医学.

更多相关视频

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

Genome-Wide CRISPR Screen for Unveiling Radiosensitive and Radioresistant Genes

Genome-Wide CRISPR Screen for Unveiling Radiosensitive and Radioresistant Genes

Published on: May 23, 2025

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...

CRISPR/Cas9 Genome Editing

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...

CRISPR and crRNAs

CRISPR and crRNAs

Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...

Homologous Recombination

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...