相关实验视频

Updated: May 23, 2025

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

Published on: March 12, 2018

在基因疗法临床试验中推进CRISPR基因组编辑:进展和未来前景

Busra Cetin¹, Fulya Erendor¹, Yunus Emre Eksi¹

¹Department of Gene and Cell Therapy, Faculty of Medicine, Akdeniz University, Antalya, Turkey.

Expert reviews in molecular medicine

|March 31, 2025

概括

基因编辑技术CRISPR提供精确的DNA修饰,用于治疗状细胞疾病等遗传疾病. 最近的FDA批准标志着一个里程碑,尽管在更广泛的应用中,安全性和交付方面的挑战仍然存在.

科学领域:

遗传学和基因组学遗传学和基因组学
生物技术是生物技术.
分子生物学分子生物学

背景情况:

基因组编辑已经取得了显著的进步,CRISPR-Cas9技术彻底改变了基因修饰.
克里斯普尔-Cas9能够精确地以RNA为导向对特定基因进行定位和编辑,并具有高精度和效率.
临床试验显示,对于诸如状细胞疾病 (SCD) 和输血依赖β-血症 (TDT) 等遗传疾病,有希望的结果.

研究的目的:

检查CRISPR技术在修改人类遗传和表观遗传代码方面的变革性作用.
为基因编辑的临床应用提供全面的重点,特别是基于CRISPR的疗法.
探索与医学中的CRISPR技术相关的潜力和挑战.

主要方法:

审查CRISPR-Cas9技术,包括基础编辑和主要编辑方法.
对基因疾病中的基因编辑进行临床试验数据的分析.
讨论正在进行的关于CRISPR在癌症,艾滋病毒和其他复杂疾病中的应用研究.

主要成果:

美国食品和药物管理局批准Casgevy,这是SCD的第一个基于CRISPR/Cas9的基因疗法,利用编辑自主CD34+造血干细胞.
展示了CRISPR技术在治疗遗传性血液疾病方面的潜力.
确定CRISPR在瘤学,病毒学和复杂疾病管理方面的新兴研究途径.

结论:

克里斯普技术代表了遗传医学的范式转变,取得了显著的临床成功.
目前正在进行的研究继续扩大基因编辑的治疗潜力.
必须解决包括非目标效应,交付,长期安全性,可扩展性和伦理考虑在内的挑战,以便广泛采用.

关键词:

这就是CRISPR/CasPR.基因编辑基因编辑基因治疗的基因疗法遗传性疾病遗传性疾病医学遗传学医学遗传学

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 CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells

Published on: July 22, 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...

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...

What is Genetic Engineering?

What is Genetic Engineering?

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...