Nano-Gene and Drug Delivery Group, Discipline of Biochemistry, University of KwaZulu-Natal, Private Bag X54001, Durban 4000, South Africa.
Int J Mol Sci. 2021 Aug 23;22(16):9082. doi: 10.3390/ijms22169082.
Neurodegenerative disorders involve the slow and gradual degeneration of axons and neurons in the central nervous system (CNS), resulting in abnormalities in cellular function and eventual cellular demise. Patients with these disorders succumb to the high medical costs and the disruption of their normal lives. Current therapeutics employed for treating these diseases are deemed palliative. Hence, a treatment strategy that targets the disease's cause, not just the symptoms exhibited, is desired. The synergistic use of nanomedicine and gene therapy to effectively target the causative mutated gene/s in the CNS disease progression could provide the much-needed impetus in this battle against these diseases. This review focuses on Parkinson's and Alzheimer's diseases, the gene/s and proteins responsible for the damage and death of neurons, and the importance of nanomedicine as a potential treatment strategy. Multiple genes were identified in this regard, each presenting with various mutations. Hence, genome-wide sequencing is essential for specific treatment in patients. While a cure is yet to be achieved, genomic studies form the basis for creating a highly efficacious nanotherapeutic that can eradicate these dreaded diseases. Thus, nanomedicine can lead the way in helping millions of people worldwide to eventually lead a better life.
神经退行性疾病涉及中枢神经系统(CNS)中轴突和神经元的缓慢而逐渐的退化,导致细胞功能异常,最终导致细胞死亡。患有这些疾病的患者承受着高昂的医疗费用和正常生活被打乱的痛苦。目前用于治疗这些疾病的疗法被认为是姑息性的。因此,人们希望有一种治疗策略能够针对疾病的原因,而不仅仅是表现出的症状。纳米医学和基因治疗的协同作用,可有效针对 CNS 疾病进展中的致病突变基因/蛋白质,为这场对抗这些疾病的战斗提供急需的动力。本文重点介绍帕金森病和阿尔茨海默病、导致神经元损伤和死亡的基因/蛋白质,以及纳米医学作为一种潜在治疗策略的重要性。在这方面已经确定了多个基因,每个基因都有不同的突变。因此,对患者进行特定治疗至关重要的是进行全基因组测序。虽然尚未找到治愈方法,但基因组研究为创建能够根除这些可怕疾病的高效纳米治疗方法奠定了基础。因此,纳米医学可以引领全球数百万人过上更好的生活。