反义寡核苷酸治疗亨廷顿氏症综述

Ariel Solis-Chiriboga, A. Bustillos
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引用次数: 0

摘要

导言:亨廷顿氏病是一种神经退行性疾病,其特征是大脑神经细胞进行性变性,目前的治疗方法主要是控制症状,因此近年来对几种疗法进行了评估,其中反义寡核苷酸是一种很有前景的策略,因为它能够调节变异亨廷蛋白的表达。方法:采用PRISMA方法在PubMed等数据库中进行文献检索,仅限于过去5年中发表的英文文献,使用的关键词包括 "亨廷顿氏病"、"反义寡核苷酸"、"治疗"、"托米纳森 "等,只选择与使用反义寡核苷酸治疗亨廷顿氏病相关的原创文章、文献综述和临床试验报告等文献。结果:共收集并分析了 150 篇文章,其中 30 篇因年代久远而被排除,40 篇因缺乏获取途径和信息质量而被排除,最后共有 80 篇文章适用了纳入标准,筛选出 25 篇文章完成了这项关于反义寡核苷酸在亨廷顿氏病中应用的文献综述:反义寡核苷酸通过直接攻击亨廷顿氏病的根本原因--变异的亨廷汀,显示出治疗亨廷顿氏病的巨大潜力。然而,目前仍存在重大挑战,因此需要更好的研究来确保这种疗法的长期疗效和安全性。
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Review of the Use of Antisense Oligonucleotides as Therapy for Huntington's Disease
Introduction: Huntington's disease is a neurodegenerative disorder characterized by the progressive degeneration of nerve cells in the brain, and whose current treatment focuses on the control of symptoms, which is why in recent years several therapies have been evaluated, including Antisense oligonucleotides a promising strategy, thanks to their ability to modulate the expression of the mutated huntingtin protein.Methods:A bibliographic search was carried out using the PRISMA methodology in databases such as PubMed, limited to documents in English published during the last 5 years, using keywords such as "Huntington's Disease", "Antisense Oligonucleotides", "Treatment" “Tominersen”, selecting only documents such as original articles, bibliographic reviews and clinical trial reports related to the use of Antisense Oligonucleotides as therapy for Huntington's Disease.Results:150 articles were collected and analyzed, of which 30 documents were excluded due to their age and 40 due to lack of access and quality of information, leaving a total of 80 articles to which inclusion criteria were applied, selecting 25 articles for completion. of this literature review on the use of antisense oligonucleotides in Huntington's diseaseConclusion: Antisense oligonucleotides show great therapeutic potential for Huntington's disease by directly attacking the underlying cause of the disease, mutated huntingtin. However, significant challenges still remain, which is why better research is needed to ensure efficacy. and safety of this therapy in the long term.
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