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Updated: Jun 16, 2025

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对CRISPR/Cas9疗法在遗传性视网膜疾病的治疗方法进行光

A B Geiger¹, J G Kennedy¹, L G Staker¹

¹Discipline of Reproduction and Development, School of Biomedicine, The University of Adelaide, Adelaide, SA, Australia; South Australian Health and Medical Research Institute (SAHMRI), Australia; Robinson Research Institute (RRI), The University of Adelaide, Adelaide, SA, Australia.

Progress in retinal and eye research

|June 7, 2025

概括

像CRISPR/Cas9这样的基因编辑技术为遗传视网膜疾病 (IRD) 提供了新的希望. 创新的输送方法正在推进这些令人失明的疾病的安全和有效治疗方法.

科学领域:

眼科医生眼科眼科
遗传学是一个遗传学.
生物技术是生物技术.

背景情况:

遗传性视网膜疾病 (IRD) 导致光受体退化,是工作年龄人群失明的主要原因.
由于其遗传基础,CRISPR/Cas9基因编辑技术为IRD提供了一个有前途的治疗途径.

研究的目的:

为IRD治疗干预进行全面审查CRISPR/Cas9技术.
分析目前用于基因编辑系统的视网膜传递方法.

主要方法:

在IRD中对CRISPR/Cas9应用的文献综述.
对纳米粒子,病毒样粒子和病毒载体传递系统的分析.
对基因编辑疗法的临床前模型的评估.

主要成果:

目前正在积极探索CRISPR/Cas9技术用于IRD治疗.
目前正在开发各种创新交付方法,以克服基因编辑系统大小和复杂性的挑战.
基因编辑和临床前模型的进步对于临床翻译至关重要.

结论:

基因编辑在管理遗传眼病方面具有变革性的潜力.
改进的输送系统和临床前模型正在使安全有效的IRD治疗更接近临床现实.

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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 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...