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Applications of adeno-associated virus vector-mediated gene delivery for neurodegenerative diseases and psychiatric diseases: Progress, advances, and challenges
Mechanisms of Ageing and Development ( IF 5.3 ) Pub Date : 2021-08-02 , DOI: 10.1016/j.mad.2021.111549
Amina Abulimiti 1 , Michael Siu-Lun Lai 1 , Raymond Chuen-Chung Chang 2
Affiliation  

Neurodegeneration is the most common disease in the elderly population due to its slowly progressive nature of neuronal deterioration, eventually leading to executive dysfunction. The pathological markers of neurological disorders are relatively well-established, however, detailed molecular mechanisms of progression and therapeutic targets are needed to develop novel treatments in human patients. Treating known therapeutic targets of neurological diseases has been aided by recent advancements in adeno-associated virus (AAV) technology. AAVs are known for their low-immunogenicity, blood-brain barrier (BBB) penetrating ability, selective neuronal tropism, stable transgene expression, and pleiotropy. In addition, the usage of AAVs has enormous potential to be optimized. Therefore, AAV can be a powerful tool used to uncover the underlying pathophysiology of neurological disorders and to increase the success in human gene therapy. This review summarizes different optimization approaches of AAV vectors with their current applications in disease modeling, neural tracing and gene therapy, hence exploring progressive mechanisms of neurodegenerative diseases as well as effective therapy. Lastly, this review discusses the limitations and future perspectives of the AAV-mediated transgene delivery system.



中文翻译:

腺相关病毒载体介导的基因传递在神经退行性疾病和精神疾病中的应用:进展、进展和挑战

神经退行性疾病是老年人群中最常见的疾病,因为其神经元退化的缓慢进行性,最终导致执行功能障碍。神经系统疾病的病理标志物相对完善,然而,需要详细的进展分子机制和治疗靶点来开发人类患者的新疗法。腺相关病毒 (AAV) 技术的最新进展有助于治疗神经系统疾病的已知治疗靶点。AAV 以其低免疫原性、血脑屏障 (BBB) 穿透能力、选择性神经元嗜性、稳定的转基因表达和多效性而闻名。此外,AAV 的使用具有巨大的优化潜力。所以,AAV 可以成为一种强大的工具,用于揭示神经系统疾病的潜在病理生理学并提高人类基因治疗的成功率。本综述总结了 AAV 载体的不同优化方法及其在疾病建模、神经追踪和基因治疗中的当前应用,从而探索神经退行性疾病的进展机制以及有效的治疗方法。最后,本综述讨论了 AAV 介导的转基因递送系统的局限性和未来前景。因此探索神经退行性疾病的进展机制以及有效的治疗方法。最后,本综述讨论了 AAV 介导的转基因递送系统的局限性和未来前景。因此探索神经退行性疾病的进展机制以及有效的治疗方法。最后,本综述讨论了 AAV 介导的转基因递送系统的局限性和未来前景。

更新日期:2021-08-10
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