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从酸盐水凝微珠中控制释放腺相关病毒,这种微珠对超声波有增强的敏感性

Shuhei Takatsuka¹, Takeshi Kubota¹, Yuta Kurashina^2,3

¹School of Integrated Design Engineering, Graduate School of Science and Technology, Keio University, Yokohama, Japan.

Biotechnology and bioengineering

|June 27, 2023

概括

这项研究引入了一种超声波触发的系统,用于控制释放腺相关病毒 (AAV) 基因疗法. 这种新的方法使用特殊的水凝微珠来实现按需的AAV输送,提高治疗的安全性和有效性.

科学领域:

生物医学工程生物医学工程
基因治疗基因治疗
材料科学材料科学材料科学

背景情况:

基于腺相关病毒 (AAV) 的基因疗法为遗传疾病治疗提供了潜力.
控制AAV释放时间对于临床应用至关重要,以防止免疫反应.
目前的方法缺乏精确控制AAV传递动力学.

研究的目的:

开发一种超声波 (US) 触发系统,用于按需发布AAV.
为了设计具有增强释放能力的酸盐水凝微珠 (AHM).
确保AAV活动在释放后保持,以确保有效的基因转染.

主要方法:

使用微滴射装置制造封装AAV和微粒 (W-MP) 的AHM.
加入W-MPs作为美国敏感释放增强剂,由于声阻变化.
用聚-l-氨酸 (PLL) 涂覆AHM以调节AAV释放配置文件.
应用美国触发局部AAV释放从工程AHMs.

主要成果:

成功制造了含有AAV和W-MP的美国响应型AHM.
根据美国的申请,对AAV的按需发布进行了证明.
通过使用释放的AAV在没有治疗活性损失的情况下成功将基因转移到细胞中.
展示了W-MPs在加强美国媒介的AAV发布中的作用.

结论:

拟议的美国触发的AAV释放系统提供了对基因疗法交付的精确时间控制.
这项技术通过管理免疫反应,提高了基于AAV的治疗的安全性和有效性.
该系统扩大了先进基因疗法应用的方法选择.

关键词:

在 AAV AAV AAV 中.药物释放释放药物释放药物释放的时间基因治疗的基因疗法水凝微珠,水凝的微珠.超声波超声波是指超声波的使用.

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