基因传递到肾脏的衣壳文库的跨物种进化

IF 28.6 1区 医学 Q1 UROLOGY & NEPHROLOGY
Monica Wang
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引用次数: 0

摘要

由于肾脏解剖和生理的复杂性,有效的基因传递到肾脏是复杂的,跨物种差异进一步阻碍了临床前研究的转化。Aravind Asokan及其同事的一项研究现在证明了腺相关病毒(AAV)文库进化对人类肾脏基因治疗发展的潜力。AAV的交付。k13和AAV。与亲代AAV9变体相比,小鼠静脉注射k20不仅导致mCherry表达更高,而且还有效地递送了程序性死亡配体1 (PD-L1)转基因,有证据表明,4周后肾脏中PD-L1表达强劲。在猪肾移植中,经输尿管逆行输送新AAV变体包装的mCherry,在同一动物体内移植后,近端小管中蛋白表达广泛,远端小管中蛋白表达水平较低。尽管病毒的生物分布没有差异,但与亲代AAV9衣壳相比,将转基因包装在新的AAV变体中,肾脏中mCherry mRNA的表达水平显著提高。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Cross-species evolution of capsid libraries for gene delivery to the kidneys

Cross-species evolution of capsid libraries for gene delivery to the kidneys
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来源期刊
Nature Reviews Nephrology
Nature Reviews Nephrology 医学-泌尿学与肾脏学
CiteScore
39.00
自引率
1.20%
发文量
127
审稿时长
6-12 weeks
期刊介绍: Nature Reviews Nephrology aims to be the premier source of reviews and commentaries for the scientific communities it serves. It strives to publish authoritative, accessible articles. Articles are enhanced with clearly understandable figures, tables, and other display items. Nature Reviews Nephrology publishes Research Highlights, News & Views, Comments, Reviews, Perspectives, and Consensus Statements. The content is relevant to nephrologists and basic science researchers. The broad scope of the journal ensures that the work reaches the widest possible audience.
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