PMID- 30458119 OWN - NLM STAT- MEDLINE DCOM- 20190401 LR - 20190401 IS - 1946-6544 (Electronic) IS - 1946-6536 (Linking) VI - 29 IP - 6 DP - 2018 Dec TI - Transparenchymal Renal Pelvis Injection of Recombinant Adeno-Associated Virus Serotype 9 Vectors Is a Practical Approach for Gene Delivery in the Kidney. PG - 251-258 LID - 10.1089/hgtb.2018.148 [doi] AB - 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. FAU - Shen, Xufeng AU - Shen X AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. AD - 4 Anhui Province PKD Center, Hefei, P.R. China. FAU - Xu, Yuchen AU - Xu Y AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. AD - 4 Anhui Province PKD Center, Hefei, P.R. China. FAU - Bai, Zhengming AU - Bai Z AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. AD - 4 Anhui Province PKD Center, Hefei, P.R. China. FAU - Ma, Dongyue AU - Ma D AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. AD - 4 Anhui Province PKD Center, Hefei, P.R. China. FAU - Niu, Qingsong AU - Niu Q AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. AD - 4 Anhui Province PKD Center, Hefei, P.R. China. FAU - Meng, Jialin AU - Meng J AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. FAU - Fan, Song AU - Fan S AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. FAU - Zhang, Li AU - Zhang L AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. FAU - Hao, Zongyao AU - Hao Z AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. FAU - Zhang, Xiansheng AU - Zhang X AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. FAU - Liang, Chaozhao AU - Liang C AD - 1 Department of Urology, the First Affiliated Hospital of Anhui Medical University, Hefei, P.R. China; Hefei, P.R. China. AD - 2 Institute of Urology and Hefei, P.R. China. AD - 3 Anhui Province Key Laboratory of Genitourinary Diseases, Anhui Medical University, Hefei, P.R. China; and Hefei, P.R. China. AD - 4 Anhui Province PKD Center, Hefei, P.R. China. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PL - United States TA - Hum Gene Ther Methods JT - Human gene therapy methods JID - 101573202 RN - 147336-22-9 (Green Fluorescent Proteins) SB - IM MH - Animals MH - Dependovirus/*genetics/metabolism MH - Epithelial Cells/metabolism MH - *Gene Transfer Techniques MH - Genetic Therapy/*methods MH - Genetic Vectors/genetics MH - Green Fluorescent Proteins/genetics/metabolism MH - Injections/methods MH - Kidney/*metabolism MH - Mice MH - Mice, Inbred C57BL OTO - NOTNLM OT - AAV OT - gene delivery OT - mouse model OT - renal disease EDAT- 2018/11/21 06:00 MHDA- 2019/04/02 06:00 CRDT- 2018/11/21 06:00 PHST- 2018/11/21 06:00 [pubmed] PHST- 2019/04/02 06:00 [medline] PHST- 2018/11/21 06:00 [entrez] AID - 10.1089/hgtb.2018.148 [doi] PST - ppublish SO - Hum Gene Ther Methods. 2018 Dec;29(6):251-258. doi: 10.1089/hgtb.2018.148.