CRISPR/Cas9基因编辑技术在阿尔茨海默病研究中的应用

Application of CRISPR/Cas9 gene-editing technology in Alzheimer disease

阿尔茨海默病是一种起病隐匿、症状逐渐加重的神经系统退行性疾病,其发病机制复杂,尚无可以逆转疾病的药物。CRISPR/Cas9技术具有周期短、细胞毒性低、价格低廉、传递简单的优点。由于CRISPR/Cas9技术可以针对不同细胞、组织或动物模型进行基因修饰,在神经退行性疾病的研究中显示出巨大的潜力。现介绍CRISPR/Cas9技术的发展、原理及其递送体系,以及目前CRISPR/Cas9技术在阿尔茨海默病模型构建、致病机制研究和靶向治疗等方面的应用与潜力,希望对相关领域的研究者起到参考作用。

Alzheimer disease(AD) is a chronic neurodegenerative disorder with insidious onset and gradual aggravation, with complex pathogenesis. At present, there is no drug to reverse the course of the disease.CRISPR/Cas9 technology has the advantages of short cycle, low cytotoxicity, low price and simple transmission.Because it can be genetically modified for different cells, tissues or animal models, it shows great potential in the study of neurodegenerative diseases. This paper introduces the development, principle and delivery system of CRISPR/Cas9 technology, as well as the application and potential of CRISPR/Cas9 technology in AD model construction, pathogenesis research and targeted therapy, hoping to play a reference role for researchers in related fields.