ArticleScientific reports2017
Kidney-specific transposon-mediated gene transfer in vivo.
Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 33 citations in OpenAlex.
- Design of Nanocarriers for Kidney Targeted Delivery of Nucleic Acid Therapeutics.Macromolecular bioscience · 2026Review
- mRNA-based therapies: current progress and future prospects for treating kidney disease.Clinical and experimental nephrology · 2026Review
- Tracking of Urine-Derived Stem Cells in Mice.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Partial correction of cystinuria type A in mice via kidney-targeted transposon delivery.Molecular therapy. Nucleic acids · 2025Article
- Urine-derived stem cells display homing, incorporation, and regeneration in human organoid and mouse models of acute kidney injury.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Precision targeting of C3+ reactive astrocyte subpopulations with endogenous ADAR in an iPSC-derived model.bioRxiv : the preprint server for biology · 2025Article
- Gene therapy and kidney diseases.Molecular therapy. Methods & clinical development · 2024Review
- mRNA as a medicine in nephrology: the future is now.Clinical kidney journal · 2023Review
- Cell and gene therapy for kidney disease.Nature reviews. Nephrology · 2023Review
- Genome Engineering of Human Urine-Derived Stem Cells to Express Lactoferrin and Deoxyribonuclease.Tissue engineering. Part A · 2023Article
- Hydrodynamic Delivery: Characteristics, Applications, and Technological Advances.Pharmaceutics · 2023Review
- Still finding ways to augment the existing management of acute and chronic kidney diseases with targeted gene and cell therapies: Opportunities and hurdles.Frontiers in medicine · 2023Review
- Transient receptor potential canonical 6 knockdown ameliorated diabetic kidney disease by inhibiting nuclear factor of activated T cells 2 expression in glomerular mesangial cells.Renal failure · 2022Article
- Efficient Messenger RNA Delivery to the Kidney Using Renal Pelvis Injection in Mice.Pharmaceutics · 2021Article
- A review of the tortuous path of nonviral gene delivery and recent progress.International journal of biological macromolecules · 2021Review
- Intravital imaging of real-time endogenous actin dysregulation in proximal and distal tubules at the onset of severe ischemia-reperfusion injury.Scientific reports · 2021Article
- Improving Molecular Therapy in the Kidney.Molecular diagnosis & therapy · 2020Review
- Review
- Effects of Tissue Pressure on Transgene Expression Characteristics via Renal Local Administration Routes from Ureter or Renal Artery in the Rat Kidney.Pharmaceutics · 2020Article
- Comparison of Gene Delivery to the Kidney by Adenovirus, Adeno-Associated Virus, and Lentiviral Vectors After Intravenous and Direct Kidney Injections.Human gene therapy · 2019Article
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 2 countries.
Funding
Abstract
Methods enabling kidney-specific gene transfer in adult mice are needed to develop new therapies for kidney disease. We attempted kidney-specific gene transfer following hydrodynamic tail vein injection using the kidney-specific podocin and gamma-glutamyl transferase promoters, but found expression primarily in the liver. In order to achieve kidney-specific transgene expression, we tested direct hydrodynamic injection of a DNA solution into the renal pelvis and found that luciferase expression was strong in the kidney and absent from extra-renal tissues. We observed heterogeneous, low-level transfection of the collecting duct, proximal tubule, distal tubule, interstitial cells, and rarely glomerular cells following injection. To assess renal injury, we performed the renal pelvis injections on uninephrectomised mice and found that their blood urea nitrogen was elevated at two days post-transfer but resolved within two weeks. Although luciferase expression quickly decreased following renal pelvis injection, the use of the piggyBac transposon system improved long-term expression. Immunosuppression with cyclophosphamide stabilised luciferase expression, suggesting immune clearance of the transfected cells occurs in immunocompetent animals. Injection of a transposon expressing erythropoietin raised the haematocrit, indicating that the developed injection technique can elicit a biologic effect in vivo. Hydrodynamic renal pelvis injection enables transposon mediated-kidney specific gene transfer in adult mice.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.