Ultrasound-guided left heart injection: a safer and more efficient strategy for mouse thoracic aortic gene delivery

IF 5.9 3区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Gene Therapy Pub Date : 2026-02-28 DOI:10.1038/s41434-026-00603-7

Yanmiao Yang, Kunyao Xu, Ya Yuan, Bo Huang, Shan Wei, Yi Zhao, Yang Song, Daxing Liu, Jianguo Liu, Dengshen Zhang

{"title":"Ultrasound-guided left heart injection: a safer and more efficient strategy for mouse thoracic aortic gene delivery","authors":"Yanmiao Yang, Kunyao Xu, Ya Yuan, Bo Huang, Shan Wei, Yi Zhao, Yang Song, Daxing Liu, Jianguo Liu, Dengshen Zhang","doi":"10.1038/s41434-026-00603-7","DOIUrl":null,"url":null,"abstract":"Thoracic aortic disease poses a significant threat due to its high mortality rates and genetic. underpinnings. While gene therapy holds promise for cures, the challenge lies in achieving. effective and safe gene delivery to the thoracic aorta. Tail vein injection (TI), is hindered by. off-target effects and hepatotoxicity, and traditional blinded percutaneous left heart injection. (TLI) carries a heightened risk of bleeding and mortality. To address these limitations. ultrasound-guided techniques present a viable solution. Adeno-associated virus (AAV) vectors. were delivered into the thoracic aorta of mice through TI, TLI, and ultrasound-guided. percutaneous left heart injection(ULI). While all three injection methods can achieve gene transduction in the thoracic aorta, the ULI approach provides the optimal balance between. high transduction efficiency and safety. The ULI method represents an efficient and safe. strategy for targeted gene delivery to the mouse thoracic aorta, providing a powerful tool for preclinical aortic gene therapy research that warrants broader application.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":"33 2","pages":"176-187"},"PeriodicalIF":5.9000,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Gene Therapy","FirstCategoryId":"3","ListUrlMain":"https://www.nature.com/articles/s41434-026-00603-7","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOCHEMISTRY & MOLECULAR BIOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Thoracic aortic disease poses a significant threat due to its high mortality rates and genetic. underpinnings. While gene therapy holds promise for cures, the challenge lies in achieving. effective and safe gene delivery to the thoracic aorta. Tail vein injection (TI), is hindered by. off-target effects and hepatotoxicity, and traditional blinded percutaneous left heart injection. (TLI) carries a heightened risk of bleeding and mortality. To address these limitations. ultrasound-guided techniques present a viable solution. Adeno-associated virus (AAV) vectors. were delivered into the thoracic aorta of mice through TI, TLI, and ultrasound-guided. percutaneous left heart injection(ULI). While all three injection methods can achieve gene transduction in the thoracic aorta, the ULI approach provides the optimal balance between. high transduction efficiency and safety. The ULI method represents an efficient and safe. strategy for targeted gene delivery to the mouse thoracic aorta, providing a powerful tool for preclinical aortic gene therapy research that warrants broader application.

Abstract Image

查看原文本刊更多论文

超声引导左心注射：一种更安全、更有效的小鼠胸主动脉基因传递策略。

胸主动脉疾病因其高死亡率和遗传性而构成重大威胁。基础。虽然基因疗法有治愈的希望，但挑战在于如何实现。有效和安全的基因输送到胸主动脉。尾静脉注射（TI）受阻于。脱靶效应和肝毒性，以及传统的盲法经皮左心注射。（TLI）会增加出血和死亡的风险。解决这些限制。超声引导技术提供了一个可行的解决方案。腺相关病毒（AAV）载体。分别通过TI、TLI和超声引导注入小鼠胸主动脉。经皮左心注射（ULI）虽然所有三种注射方法都可以在胸主动脉中实现基因转导，但ULI入路提供了最佳的平衡。转导效率高，安全性好。ULI方法是一种高效、安全的方法。小鼠胸主动脉靶向基因递送策略，为临床前主动脉基因治疗研究提供了强有力的工具，值得广泛应用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Gene Therapy 医学-生化与分子生物学

CiteScore

9.70

自引率

2.00%

发文量

审稿时长

4-8 weeks

期刊介绍： Gene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine.