临床内耳基因治疗的新型传递方法

IF 16.4 1区化学 Q1 CHEMISTRY, MULTIDISCIPLINARY

Accounts of Chemical Research Pub Date : 2024-11-06 DOI:10.1097/MAO.0000000000004366

Long Long Zhang, JingHan Wang, Zi Wen Gao, Jun Lv, Luo Ying Jiang, Chong Cui, Zi Jing Wang, Da Qi Wang, Yu Xin Chen, Xin Tai Fan, Cheng Ye, Hui Wang, Bing Chen, Wu Qing Wang, Hua Wei Li, Yi Lai Shu

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引用次数: 0

摘要

背景：为了创建和开发临床内耳基因治疗的给药方法，我们通过经内窥镜耳科手术（TEES）进行了一项使用移液微针的经圆窗膜（RWM）微注射研究：方法：实施经圆窗膜显微注射手术涉及七具尸体标本，并对手术程序和移液微针进行了开发和优化。TEES 手术包括鼓室显像、RWM 暴露、镫骨脚板穿孔和经 RWM 显微注射。在显微注射过程中，对不同移液微针的可行性进行了评估：结果：在 TEES 中很容易暴露 RWM 显微注射部位，软连接移液微针最适合经 RWM 显微注射。在微注射过程中可以明显观察到液体从镫骨穿孔处流出，这表明内耳给药是成功的。这种内耳给药方法已成功应用于临床试验：结论：通过 TEES 中的软连接移液微针进行经 RWM 显微注射被证明是一种可行的内耳基因治疗给药方法。

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A Novel Delivery Approach of Clinical Inner Ear Gene Therapy.

Background: To create and develop a delivery approach for clinical inner ear gene therapy, we conducted a study of trans-round window membrane (RWM) microinjection using a pipetting microneedle via transcanal endoscopic ear surgery (TEES).

Methods: The implementation of the trans-RWM microinjection surgery involved seven cadaveric specimens, and the surgical procedures and the pipetting microneedle were developed and optimized. The TEES procedures included tympanic cavity visualization, RWM exposure, stapes footplate perforation, and trans-RWM microinjection. The feasibility of different pipetting microneedles was evaluated during microinjection.

Results: Exposure of the RWM microinjection site could be easily achieved in TEES, and the soft-connected pipetting microneedle was most suitable for the trans-RWM microinjection. The fluid outflow from stapes perforation could be visibly observed during the microinjection, which indicated inner ear drug delivery was successful. This inner ear drug delivery approach was successfully applied in the clinical trial.

Conclusion: The trans-RWM microinjection via the soft-connected pipetting microneedle in TEES was proved to be a feasible delivery approach of the inner ear gene therapy.

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来源期刊

Accounts of Chemical Research 化学-化学综合

CiteScore

31.40

自引率

1.10%

发文量

312

审稿时长

2 months

期刊介绍： Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.