{"title":"CRISPR/Cas9 Gene Editing: A Novel Approach Towards Alzheimer's Disease Treatment.","authors":"Siddhant Tripathi, Yashika Sharma, Rajesh Rane, Dileep Kumar","doi":"10.2174/0118715273283786240408034408","DOIUrl":null,"url":null,"abstract":"<p><p>In defiance of the vast amount of information regarding Alzheimer's disease (AD) that has been learned over the past thirty years, progress toward developing an effective therapy has been difficult. A neurological ailment that progresses and cannot be reversed is Alzheimer's disease, which shows neurofibrillary tangles, beta-amyloid plaque, and a lack of cognitive processes that is created by tau protein clumps with hyperphosphorylation that finally advances to neuronal damage without a recognized treatment, which has stimulated research into new therapeutic strategies. The protein CAS9 is linked to CRISPR, which is a clustered Regularly Interspaced Short Palindromic Repeat that inactivates or corrects a gene by recognizing a gene sequence that produces a doublestranded break has enchanted a whole amount of interest towards its potency to cure gene sequences in AD. The novel CRISPR-Cas9 applications for developing <i>in vitro</i> and <i>in vivo</i> models to the benefit of AD investigation and therapies are thoroughly analyzed in this work. The discussion will also touch on the creation of delivery methods, which is a significant obstacle to the therapeutic use of CRISPR/Cas9 technology. By concentrating on specific genes, such as those that are significant early- onset AD risk factors and late-onset AD risk factors, like the apolipoprotein E4 (APOE4) gene, this study aims to evaluate the potential application of CRISPR/Cas9 as a possible treatment for AD.</p>","PeriodicalId":93947,"journal":{"name":"CNS & neurological disorders drug targets","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"CNS & neurological disorders drug targets","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2174/0118715273283786240408034408","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
Abstract
In defiance of the vast amount of information regarding Alzheimer's disease (AD) that has been learned over the past thirty years, progress toward developing an effective therapy has been difficult. A neurological ailment that progresses and cannot be reversed is Alzheimer's disease, which shows neurofibrillary tangles, beta-amyloid plaque, and a lack of cognitive processes that is created by tau protein clumps with hyperphosphorylation that finally advances to neuronal damage without a recognized treatment, which has stimulated research into new therapeutic strategies. The protein CAS9 is linked to CRISPR, which is a clustered Regularly Interspaced Short Palindromic Repeat that inactivates or corrects a gene by recognizing a gene sequence that produces a doublestranded break has enchanted a whole amount of interest towards its potency to cure gene sequences in AD. The novel CRISPR-Cas9 applications for developing in vitro and in vivo models to the benefit of AD investigation and therapies are thoroughly analyzed in this work. The discussion will also touch on the creation of delivery methods, which is a significant obstacle to the therapeutic use of CRISPR/Cas9 technology. By concentrating on specific genes, such as those that are significant early- onset AD risk factors and late-onset AD risk factors, like the apolipoprotein E4 (APOE4) gene, this study aims to evaluate the potential application of CRISPR/Cas9 as a possible treatment for AD.
尽管过去三十年来人们了解了大量有关阿尔茨海默病(AD)的信息,但在开发有效疗法方面却一直举步维艰。阿尔茨海默病是一种进展缓慢且无法逆转的神经系统疾病,它表现为神经纤维缠结、β-淀粉样蛋白斑块和认知过程缺失,而认知过程缺失是由具有高磷酸化的 tau 蛋白团块造成的,最终发展为神经元损伤,却没有公认的治疗方法,这激发了人们对新治疗策略的研究。CAS9蛋白与CRISPR相关联,CRISPR是一种簇状正则间隔短回文重复序列(clustered Regularly Interspaced Short Palindromic Repeat),它通过识别产生双链断裂的基因序列来使基因失活或纠正基因。本研究将深入分析 CRISPR-Cas9 在开发体外和体内模型方面的新应用,以促进 AD 研究和治疗。讨论还将涉及传递方法的创建,这是 CRISPR/Cas9 技术用于治疗的一个重大障碍。通过集中研究特定基因,如那些重要的早发性AD风险因素和晚发性AD风险因素,如载脂蛋白E4(APOE4)基因,本研究旨在评估CRISPR/Cas9作为一种可能的AD治疗方法的潜在应用。