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Current viral-mediated gene transfer research for treatment of Alzheimer's disease.
Biotechnology and Genetic Engineering Reviews ( IF 3.2 ) Pub Date : 2018-10-14 , DOI: 10.1080/02648725.2018.1523521
Andrew Octavian Sasmita 1
Affiliation  

Alzheimer's disease (AD) is the most common form of dementia and has affected millions of individuals worldwide. The hallmarks of AD include the amyloid beta plaque deposits, tau neurofibrillary tangles, altered neuronal signaling, alongside decline in memory and cognitive functions. Conventional drug therapies do exist, such as donepezil or aducanumab, but these drugs mostly focus on halting AD progression instead of causing a reversal within the disease. In an effort to ameliorate and ultimately cure AD, researchers have delved into viral-mediated gene therapy to fix this disease from its root molecular causes. To date, adeno-associated virus and lentiviral vectors have remained the most vastly studied among other viral vectors to combat AD. These vectors could be employed alongside various genetic materials based on the types of processes we want to alter to yield a positive effect, such as disruption of amyloidogenic pathway, neuroprotection and lipid metabolism pathways. Recent studies and trials were reviewed in this article, highlighting their clinical significance, differences and limitations between each method. By learning from the different combinations and possibilities of viral-mediated gene transfer, researchers would then get a step closer in ameliorating symptoms and possibly in curing AD.

中文翻译:

目前用于治疗阿尔茨海默氏病的病毒介导的基因转移研究。

阿尔茨海默氏病(AD)是痴呆症最常见的形式,已经影响了全球数百万的人。AD的特征包括淀粉样蛋白β斑块沉积,tau神经原纤维缠结,神经元信号改变,记忆力和认知功能下降。确实存在常规药物疗法,例如多奈哌齐或阿杜那单抗,但这些药物主要集中在阻止AD进展,而不是引起疾病内的逆转。为了改善和最终治愈AD,研究人员已深入研究病毒介导的基因疗法,以从根源上解决该疾病。迄今为止,腺相关病毒和慢病毒载体仍是抗击AD的其他病毒载体中研究最广泛的。根据我们想要改变以产生积极作用的过程类型,可以将这些载体与各种遗传物质一起使用,例如破坏淀粉样蛋白生成途径,神经保护和脂质代谢途径。本文对最近的研究和试验进行了综述,强调了它们的临床意义,每种方法之间的差异和局限性。通过从病毒介导的基因转移的不同组合和可能性中学习,研究人员将在改善症状和可能治愈AD方面走近一步。
更新日期:2020-03-07
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