Transparenchymal Renal Pelvis Injection of Recombinant Adeno-Associated Virus Serotype 9 Vectors Is a Practical Approach for Gene Delivery in the Kidney.

Q1 Immunology and Microbiology

Human Gene Therapy Methods Pub Date : 2018-12-01 DOI:10.1089/hgtb.2018.148

Xufeng Shen, Yuchen Xu, Zhengming Bai, Dongyue Ma, Qingsong Niu, Jialin Meng, Song Fan, Li Zhang, Zongyao Hao, Xiansheng Zhang, Chaozhao Liang

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引用次数: 9

Abstract

Gene therapy has great potential in treating human diseases, but little progress has been made in preclinical and clinical studies of renal diseases. To find an effective gene delivery approach in the kidney, transparenchymal renal pelvis injection was developed. Using adeno-associated virus serotype 9 (AAV9) vectors, the gene delivery efficiency and safety of this administration method were evaluated. The results showed that the exogenous gene was expressed in the tubular epithelial cells of the injected kidney, with a much lower expression level in the contralateral kidney. Extra-renal transduction in the liver was also observed in this study, with the liver function of AAV9-injected mice comparable to that of control mice. Altogether, the administration of AAV9 vectors by newly established transparenchymal renal pelvis injection achieved the desired exogenous gene expression in renal tubular cells, and hence might be one possible way for gene therapy in renal diseases.

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透明腔肾盂注射重组腺相关病毒血清型9载体是肾脏基因传递的一种实用方法。

基因治疗在治疗人类疾病方面具有巨大的潜力，但在肾脏疾病的临床前和临床研究方面进展甚微。为了寻找一种有效的基因在肾脏中的传递途径，透明腔肾盂注射被开发出来。采用腺相关病毒血清型9 (AAV9)载体，评价该给药方法的基因传递效率和安全性。结果表明，外源基因在注射肾小管上皮细胞中表达，在对侧肾中的表达水平低得多。本研究还观察到肝脏的肾外转导，注射aav9小鼠的肝功能与对照组小鼠相当。总之，通过新建立的透明肾盂注射给药AAV9载体在肾小管细胞中实现了所需的外源基因表达，因此可能是肾脏疾病基因治疗的一种可能方法。

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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来源期刊

Human Gene Therapy Methods BIOTECHNOLOGY & APPLIED MICROBIOLOGY-GENETICS & HEREDITY

CiteScore

5.80

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases. The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.