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Mini-Review Article

DREADD病毒载体用于中枢神经系统的治疗

卷 21, 期 3, 2021

发表于: 11 February, 2021

页: [191 - 206] 页: 16

弟呕挨: 10.2174/1566523221666210211102435

价格: $65

摘要

设计药物特异性激活的设计受体(DREADD)是基因修饰的g蛋白偶联受体(GPCRs)，它可以被合成配体激活，否则在内源性受体上是惰性的。DREADD可以在中枢神经系统(CNS)的细胞中表达，随后当合成配体被系统管理时，为远程和可逆地沉默或激活靶细胞提供了机会。在神经科学领域，DREADD迄今已被证明是几个研究领域的有用工具，并为神经疾病的基因治疗策略的发展提供了相当大的潜力。然而，为了设计一种基于DREADD的基因治疗，有必要首先评估文献中用于递送这些化学发生工具的病毒载体递送方法。本文评估了目前用于DREADD交付的每一项主要战略，讨论了它们各自的优点和局限性。我们关注基于腺相关病毒(AAV)和基于慢病毒的系统，以及通过细胞类型特异性启动子和假分型来操纵这些系统。此外，我们还讨论了如何改进病毒介导的DREADD递送，使其成为一种可行的基因治疗策略，从而扩大其翻译潜力。

关键词: DREADD，化学遗传学，AAV，慢病毒，基因治疗，中枢神经系统，病毒载体

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图形摘要

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