Gene therapy for the heart: encapsulated viruses to the rescue

Uma Maheswari Deshetty, S. Sil, Shilpa Buch
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引用次数: 0

Abstract

This commentary provides an in-depth analysis and perspective on the pioneering research article titled ‘Extracellular Vesicle-Encapsulated Adeno-Associated Viruses for Therapeutic Gene Delivery to the Heart’. The original study explores the innovative use of extracellular vesicle-encapsulated AAVs (EV-AAV-6 and -9) as a superior gene-delivery approach for cardiomyocytes (CMs), which not only provides increased AAV neutralizing antibody (NAb) resistance but also has implications for increased gene delivery efficacy to ischemic hearts. This study examined the efficacy of EVs isolated from the conditioned medium of AAV-6 and -9 producing HEK293T cells in combinatorial in vitro and in vivo model systems in comparison to free AAVs in the presence of the NAbs. This commentary highlights the key findings, discusses potential implications, limitations, and suggests future directions for research in this evolving field.
心脏基因疗法:封装病毒的救星
这篇评论深入分析和透视了题为 "细胞外囊泡包裹的腺相关病毒用于治疗性基因递送到心脏 "的开创性研究文章。该原创性研究探讨了细胞外囊泡包封的 AAV(EV-AAV-6 和 -9)作为心肌细胞(CMs)基因递送方法的创新应用,它不仅能增强 AAV 中和抗体(NAb)的抗性,而且对提高缺血心脏的基因递送效果具有重要意义。本研究考察了从产生 AAV-6 和 -9 的 HEK293T 细胞的条件培养基中分离出的 EVs 在体外和体内组合模型系统中与存在 NAb 的游离 AAVs 相比的功效。这篇评论强调了主要发现,讨论了潜在的影响和局限性,并为这一不断发展的领域提出了未来的研究方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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