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CRISPR-Cas 系统在自身免疫性疾病中的研究进展。

Current progress in CRISPR-Cas systems for autoimmune diseases.

机构信息

Institute of Biosciences and Technology, Shri Ramswaroop Memorial University, Lucknow, Barabanki, Uttar Pradesh, India.

Department of Biotechnology, Faculty of Engineering and Technology, Rama University, Mandhana, Kanpur, Uttar Pradesh, India.

出版信息

Prog Mol Biol Transl Sci. 2024;208:231-259. doi: 10.1016/bs.pmbts.2024.07.011. Epub 2024 Aug 14.

Abstract

A body develops an autoimmune illness when its immune system mistakenly targets healthy cells and organs. Eight million people are affected by more than 80 autoimmune diseases. The public's and individuals' well-being is put at risk. Type 1 diabetes, lupus, rheumatoid arthritis, and multiple sclerosisare autoimmune diseases. Tissue injury, nociceptive responses, and persistent inflammation are the results of these stresses. Concerns about healthcare costs, health, and physical limitations contribute to these issues. Given their prevalence, it is crucial to enhance our knowledge, conduct thorough research, and provide all-encompassing support to women dealing with autoimmune diseases. This will lead to better public health and better patient outcomes. Most bacteria's immune systems employ CRISPR-Cas, a state-of-the-art technique for editing genes. For Cas to break DNA with pinpoint accuracy, a guide RNA employs a predetermined enzymatic pathway. Genetic modifications started. After it was developed, this method was subjected to much research on autoimmune diseases. By modifying immune pathways, CRISPR gene editing can alleviate symptoms, promote immune system tolerance, and decrease autoimmune reactivity. The autoimmune diseases that CRISPR-Cas9 targets now have no treatment or cure. Results from early clinical trials and preclinical studies of autoimmune medicines engineered using CRISPR showed promise. Modern treatments for rheumatoid arthritis,multiple sclerosis, and type 1 diabetes aim to alter specific genetic or immune mechanisms. Accurate CRISPR editing can fix autoimmune genetic disorders. Modifying effector cells with CRISPR can decrease autoimmune reactions. These cells include cytotoxic T and B lymphocytes. Because of improvements in delivery techniques and kits, CRISPR medications are now safer, more effective, and more accurately targeted. It all comes down to intricate immunological reactions and unexpected side consequences. Revolutionary cures for autoimmune problems and highly personalized medical therapies have been made possible by recent advancements in CRISPR.

摘要

当人体的免疫系统错误地攻击健康细胞和器官时,就会患上自身免疫性疾病。有 800 万人受到 80 多种自身免疫性疾病的影响。公众和个人的健康受到威胁。1 型糖尿病、狼疮、类风湿关节炎和多发性硬化症等都是自身免疫性疾病。这些压力会导致组织损伤、疼痛反应和持续的炎症。对医疗保健成本、健康和身体限制的担忧也是导致这些问题的原因之一。鉴于它们的普遍性,提高我们的知识水平,进行彻底的研究,并为患有自身免疫性疾病的女性提供全面的支持至关重要。这将有助于改善公众健康和患者的治疗效果。大多数细菌的免疫系统都采用了 CRISPR-Cas,这是一种基因编辑的先进技术。为了使 Cas 以极高的精度切割 DNA,向导 RNA 采用了一种预先确定的酶促途径。遗传修饰开始了。该方法开发后,在自身免疫性疾病方面进行了大量研究。通过修饰免疫途径,CRISPR 基因编辑可以缓解症状、促进免疫系统耐受和减少自身免疫反应。目前针对 CRISPR-Cas9 的自身免疫性疾病尚无治疗或治愈方法。使用 CRISPR 工程设计的自身免疫药物的早期临床试验和临床前研究结果显示出了希望。针对类风湿关节炎、多发性硬化症和 1 型糖尿病的现代治疗方法旨在改变特定的遗传或免疫机制。精确的 CRISPR 编辑可以修复自身免疫性遗传疾病。使用 CRISPR 修饰效应细胞可以减少自身免疫反应。这些细胞包括细胞毒性 T 和 B 淋巴细胞。由于递送技术和试剂盒的改进,CRISPR 药物现在更安全、更有效、更有针对性。所有这一切都归结于复杂的免疫反应和意想不到的副作用。CRISPR 的最新进展为自身免疫问题的革命性治疗方法和高度个性化的医疗疗法提供了可能。

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