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Delivery of neurotrophic factors in the treatment of age-related chronic neurodegenerative diseases

机译:治疗年龄相关慢性神经变性疾病的神经营养因子

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Introduction: Neurodegenerative diseases are those wherein the neurons in the brain or peripheral nervous system lose their function, eventually culminating in neuronal death. Aging acts as the predominant factor here due to the reduced protein turnover rate in aging cells. As neurotrophic factors possess imperative roles in protecting the neurons and restoring their functionality, design of different modalities to deliver them to the brain would significantly enhance the therapeutic benefits. Areas covered: This review covers the various mechanisms of neurodegeneration, its molecular link with aging, different neurotrophic factor classes and their potentials, current treatment strategies, the challenges associated with the delivery of neurotrophic factors, administration routes, design of different delivery vehicle design, alternative modalities of delivery, and the clinical translational challenges of these strategies. Expert opinion: A deeper molecular level understanding about the complexity of neurodegeneration, discovery of potential biomarkers, which helps identifying the right targets, finding the accurate animal model completely recapitulating the human scenario, and a validated design of clinical trials would immensely help in overcoming the present challenges. The substantial developments in the field of gene therapy, usage of small molecules and peptide mimetics, combinatory approaches, etc. definitely give brighter hopes.
机译:介绍:神经退行性疾病是那些脑或外周神经系统中神经元失去其功能的疾病,最终终止于神经元死亡。由于老化细胞中的蛋白质周转率降低,因此衰老作为占主导地位因子。由于神经营养因素具有保护神经元并恢复其功能的必要作用,因此将不同的方式设计为将其传递给大脑的设计将显着提高治疗益处。涵盖的区域:本综述涵盖了神经变性的各种机制,其分子环与老化,不同的神经营养因子类别及其潜力,当前的治疗策略,与递送神经营养因素,行政路线,不同送货车设计的挑战,交付的替代方式,以及这些策略的临床平移挑战。专家意见:一种深入的分子水平,了解神经变性的复杂性,发现潜在的生物标志物,这有助于识别正确的目标,找到完全重新承载人类情景的准确动物模型,以及临床试验的验证设计将大大帮助克服克服呈现挑战。基因治疗领域的实质性发展,使用小分子和肽模拟物,组合方法等肯定是更亮的希望。

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