利用工程病毒载体的帕金森病下一代基因治疗。
Next-Generation Gene Therapy for Parkinson's Disease Using Engineered Viral Vectors.
机构信息
Molecular Neuromodulation, Wallenberg Neuroscience Center, Lund University, Lund, Sweden.
出版信息
J Parkinsons Dis. 2021;11(s2):S209-S217. doi: 10.3233/JPD-212674.
Abstract
Recent technological and conceptual advances have resulted in a plethora of exciting novel engineered adeno associated viral (AAV) vector variants. They all have unique characteristics and abilities. This review summarizes the development and their potential in treating Parkinson's disease (PD). Clinical trials in PD have shown over the last decade that AAV is a safe and suitable vector for gene therapy but that it also is a vehicle that can benefit significantly from improvement in specificity and potency. This review provides a concise collection of the state-of-the-art for synthetic capsids and their utility in PD. We also summarize what therapeutical strategies may become feasible with novel engineered vectors, including genome editing and neuronal rejuvenation.
摘要
近年来，技术和概念上的进步带来了大量令人兴奋的新型工程腺相关病毒（AAV）载体变体。它们都具有独特的特点和能力。这篇综述总结了它们在治疗帕金森病（PD）方面的发展和潜力。过去十年的 PD 临床试验表明，AAV 是一种安全且合适的基因治疗载体，但它也可以通过提高特异性和效力而显著受益。本综述提供了一个关于合成衣壳及其在 PD 中的应用的最新技术的简明集合。我们还总结了新型工程载体可能实现的治疗策略，包括基因组编辑和神经元再生。
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