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Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications
Advanced Drug Delivery Reviews ( IF 15.2 ) Pub Date : 2024-06-19 , DOI: 10.1016/j.addr.2024.115363 Dezhuang Ye 1 , Chinwendu Chukwu 1 , Yaoheng Yang 1 , Zhongtao Hu 1 , Hong Chen 2
Advanced Drug Delivery Reviews ( IF 15.2 ) Pub Date : 2024-06-19 , DOI: 10.1016/j.addr.2024.115363 Dezhuang Ye 1 , Chinwendu Chukwu 1 , Yaoheng Yang 1 , Zhongtao Hu 1 , Hong Chen 2
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Adeno-associated virus (AAV) vectors have emerged as a promising tool in the development of gene therapies for various neurological diseases, including Alzheimer’s disease and Parkinson’s disease. However, the blood–brain barrier (BBB) poses a significant challenge to successfully delivering AAV vectors to the brain. Strategies that can overcome the BBB to improve the AAV delivery efficiency to the brain are essential to successful brain-targeted gene therapy. This review provides an overview of existing strategies employed for AAV delivery to the brain, including direct intraparenchymal injection, intra-cerebral spinal fluid injection, intranasal delivery, and intravenous injection of BBB-permeable AAVs. Focused ultrasound has emerged as a promising technology for the noninvasive and spatially targeted delivery of AAV administered by intravenous injection. This review also summarizes each strategy’s current preclinical and clinical applications in treating neurological diseases. Moreover, this review includes a detailed discussion of the recent advances in the emerging focused ultrasound-mediated AAV delivery. Understanding the state-of-the-art of these gene delivery approaches is critical for future technology development to fulfill the great promise of AAV in neurological disease treatment.
中文翻译：
腺相关病毒载体递送至大脑：技术进步和临床应用
腺相关病毒（AAV）载体已成为开发各种神经系统疾病（包括阿尔茨海默病和帕金森病）基因疗法的有前途的工具。然而，血脑屏障（BBB）对成功将 AAV 载体传递到大脑提出了重大挑战。克服血脑屏障以提高 AAV 向大脑的递送效率的策略对于成功的脑靶向基因治疗至关重要。本综述概述了将 AAV 递送至大脑的现有策略，包括直接脑实质内注射、脑脊液内注射、鼻内递送和静脉注射 BBB 渗透性 AAV。聚焦超声已成为一种有前景的技术，用于通过静脉注射进行 AAV 的无创和空间靶向递送。这篇综述还总结了每种策略目前在治疗神经系统疾病方面的临床前和临床应用。此外，本综述还详细讨论了新兴的聚焦超声介导的 AAV 递送的最新进展。了解这些基因传递方法的最新技术对于未来技术发展以实现 AAV 在神经系统疾病治疗中的巨大前景至关重要。
更新日期：2024-06-19
中文翻译：
腺相关病毒载体递送至大脑：技术进步和临床应用
腺相关病毒（AAV）载体已成为开发各种神经系统疾病（包括阿尔茨海默病和帕金森病）基因疗法的有前途的工具。然而，血脑屏障（BBB）对成功将 AAV 载体传递到大脑提出了重大挑战。克服血脑屏障以提高 AAV 向大脑的递送效率的策略对于成功的脑靶向基因治疗至关重要。本综述概述了将 AAV 递送至大脑的现有策略，包括直接脑实质内注射、脑脊液内注射、鼻内递送和静脉注射 BBB 渗透性 AAV。聚焦超声已成为一种有前景的技术，用于通过静脉注射进行 AAV 的无创和空间靶向递送。这篇综述还总结了每种策略目前在治疗神经系统疾病方面的临床前和临床应用。此外，本综述还详细讨论了新兴的聚焦超声介导的 AAV 递送的最新进展。了解这些基因传递方法的最新技术对于未来技术发展以实现 AAV 在神经系统疾病治疗中的巨大前景至关重要。
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