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Delivery of neurotrophic factors in the treatment of age-related chronic neurodegenerative diseases.
Expert Opinion on Drug Delivery ( IF 6.6 ) Pub Date : 2020-02-14 , DOI: 10.1080/17425247.2020.1727443
Smrithi Padmakumar 1 , Maie S Taha 1, 2, 3 , Ekta Kadakia 1, 4 , Benjamin S Bleier 2 , Mansoor M Amiji 1
Affiliation  

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.

中文翻译:

神经营养因子的递送,用于治疗与年龄相关的慢性神经退行性疾病。

简介:神经退行性疾病是指大脑或周围神经系统中的神经元失去功能,最终导致神经元死亡的疾病。在这里,衰老是主要的因素,因为衰老细胞中的蛋白质更新率降低。由于神经营养因子在保护神经元和恢复神经元功能方面起着至关重要的作用,因此设计不同的方式将其递送至大脑将显着增强治疗效果。研究范围:本综述涵盖了神经退行性变的各种机制,其与衰老的分子联系,不同的神经营养因子类别及其潜力,当前的治疗策略,与神经营养因子输送相关的挑战,给药途径,不同运载工具设计的设计,替代的给药方式,以及这些策略的临床翻译挑战。专家意见:对神经变性的复杂性有更深入的分子水平了解,发现潜在的生物标记物,有助于识别正确的靶标,找到准确的动物模型,全面再现人类的情况,经过验证的临床试验设计将极大地帮助克服当前的挑战。基因治疗,小分子和肽模拟物的使用,组合方法等领域的重大发展无疑带来了更广阔的希望。这有助于确定正确的目标,找到可以完全概括人类情况的准确动物模型,以及经过验证的临床试验设计将极大地帮助克服当前的挑战。基因治疗,小分子和肽模拟物的使用,组合方法等领域的重大发展无疑带来了更广阔的希望。这有助于确定正确的目标,找到可以完全概括人类情况的准确动物模型,以及经过验证的临床试验设计将极大地帮助克服当前的挑战。基因治疗,小分子和肽模拟物的使用,组合方法等领域的重大发展无疑带来了更广阔的希望。
更新日期:2020-03-22
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