使用锌指核酸酶 (ZFNs) 的基因组编辑方法治疗运动神经元疾病。

IF 2.2 4区 医学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY
Medisetti Manikishore, Sandeep Kumar Maurya, Sunny Rathee, Umesh Kumar Patil
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引用次数: 0

摘要

运动神经元疾病是一种可通过遗传代代相传的疾病,也可因基因自发突变而发生。这些疾病会削弱运动神经元和肌肉之间的联系,从而导致神经元和肌肉之间的协调受到干扰,进而使动作变得异常,全世界每年有数百万人罹患这些不同类型的运动神经元疾病。到目前为止,还没有治疗这类疾病的适当方法,但人们正在积极开展研究工作,以永久性地治疗这些疾病。一些基因疗法在治疗这些疾病方面取得了可喜的成果,具体来说,基因修饰技术是其中的佼佼者,许多类型的核酸酶正在用功能性核酸酶取代变异基因。锌指核酸酶(ZFNs)是其中之一,具有良好的疾病治疗潜力,效果准确而理想。在这篇综述中,我们将全面介绍锌指核酸酶及其缺点,以及它们在基因疗法中的未来前景,并将很快介绍其他类型的核酸酶介导的基因疗法。此外,我们还指出了影响基因疗法的一些因素,并提供了一些详细信息。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Genome Editing Approaches Using Zinc Finger Nucleases (ZFNs) for the Treatment of Motor Neuron Diseases.

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.

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来源期刊
Current pharmaceutical biotechnology
Current pharmaceutical biotechnology 医学-生化与分子生物学
CiteScore
5.60
自引率
3.60%
发文量
203
审稿时长
6 months
期刊介绍: Current Pharmaceutical Biotechnology aims to cover all the latest and outstanding developments in Pharmaceutical Biotechnology. Each issue of the journal includes timely in-depth reviews, original research articles and letters written by leaders in the field, covering a range of current topics in scientific areas of Pharmaceutical Biotechnology. Invited and unsolicited review articles are welcome. The journal encourages contributions describing research at the interface of drug discovery and pharmacological applications, involving in vitro investigations and pre-clinical or clinical studies. Scientific areas within the scope of the journal include pharmaceutical chemistry, biochemistry and genetics, molecular and cellular biology, and polymer and materials sciences as they relate to pharmaceutical science and biotechnology. In addition, the journal also considers comprehensive studies and research advances pertaining food chemistry with pharmaceutical implication. Areas of interest include: DNA/protein engineering and processing Synthetic biotechnology Omics (genomics, proteomics, metabolomics and systems biology) Therapeutic biotechnology (gene therapy, peptide inhibitors, enzymes) Drug delivery and targeting Nanobiotechnology Molecular pharmaceutics and molecular pharmacology Analytical biotechnology (biosensing, advanced technology for detection of bioanalytes) Pharmacokinetics and pharmacodynamics Applied Microbiology Bioinformatics (computational biopharmaceutics and modeling) Environmental biotechnology Regenerative medicine (stem cells, tissue engineering and biomaterials) Translational immunology (cell therapies, antibody engineering, xenotransplantation) Industrial bioprocesses for drug production and development Biosafety Biotech ethics Special Issues devoted to crucial topics, providing the latest comprehensive information on cutting-edge areas of research and technological advances, are welcome. Current Pharmaceutical Biotechnology is an essential journal for academic, clinical, government and pharmaceutical scientists who wish to be kept informed and up-to-date with the latest and most important developments.
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