Suppr超能文献

使用锌指核酸酶(ZFNs)治疗运动神经元疾病的基因组编辑方法

Genome Editing Approaches Using Zinc Finger Nucleases (ZFNs) for the Treatment of Motor Neuron Diseases.

作者信息

Manikishore Medisetti, Maurya Sandeep Kumar, Rathee Sunny, Patil Umesh Kumar

机构信息

Department of Pharmaceutical Sciences, Dr. Harisingh Gour Vishwavidyalaya (A Central University), Sagar (M.P.) 470003, India.

出版信息

Curr Pharm Biotechnol. 2024 Jun 6. doi: 10.2174/0113892010307288240526071810.

Abstract

Motor neuron disorders are diseases that can be passed through generations by heredity or they occur due to spontaneous mutations in the gene. These are the disorders that weaken the connection between motor neurons and the muscles, due to this the coordination between the neurons and muscles gets disturbed and thereby the actions become abnormal, every year millions of people around the world suffer from these different types of motor neuron disorders. Till now there is no proper known treatment for this type of disorder, there is active research work going on to treat these diseases permanently. Some gene therapy treatments are giving promising results in the treatment of these diseases, specifically, genetic modification techniques are the front liners, and many types of nucleases are doing their work to replace the mutated gene with a functional one. Zinc finger nucleases (ZFNs) are one of them with good disease treatment potential with accurate and desirable effects. In this review, we note the complete information about ZFNs and their drawbacks along with their future prospective in gene therapy and also shortly with other types of nucleases-mediated gene therapies. There also some factors that influence the gene therapy treatment are also noted along with some detailed information.

摘要

运动神经元疾病是一类可通过遗传代代相传的疾病,或者是由于基因的自发突变而发生的疾病。这些疾病会削弱运动神经元与肌肉之间的联系,因此神经元与肌肉之间的协调受到干扰,从而导致行动异常,全世界每年有数百万人患有这些不同类型的运动神经元疾病。到目前为止,这类疾病尚无确切已知的治疗方法,目前正在进行积极的研究工作以永久治疗这些疾病。一些基因治疗方法在治疗这些疾病方面取得了有前景的结果,具体而言,基因编辑技术处于前沿,多种核酸酶正在发挥作用,用功能正常的基因替换突变基因。锌指核酸酶(ZFNs)就是其中之一,具有良好的疾病治疗潜力,效果准确且理想。在这篇综述中,我们记录了关于锌指核酸酶的完整信息及其缺点,以及它们在基因治疗中的未来前景,同时也简要介绍了其他类型的核酸酶介导的基因治疗。还记录了一些影响基因治疗的因素以及一些详细信息。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验