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Updated: Jul 6, 2026

Effective and Safe Gene Delivery to the Mouse Kidney via Slow Retrograde Renal Pelvis Injection of Adeno-Associated Virus Vectors
Published on: August 1, 2025
Gene therapy for acute renal failure
Juan Torras1, Josep M Cruzado, Immaculada Herrero-Fresneda
1Nephrology Service and Laboratory of Experimental Nephrology, Hospital Universitari de Bellvitge, University of Barcelona, IDIBELL, Barcelona, Spain.
Abstract:
Thanks to the progressive understanding of the cellular and molecular basis of renal function and disease, during the next several decades new therapeutic approaches to a wide range of kidney disorders, including acute renal failure (ARF), will be developed. In this regard, the repair of ischemic and toxic ARF is critically dependent on a redundant, interactive cytokine and growth factors network to return kidney function to near-normal baseline function. A newer strategy in biotechnology is the development of recombinant genetic engineered compounds and, recently, cell therapy derivatives. Gene therapy offers a novel approach for prevention and treatment of renal diseases. Technical advances in viral vector systems and the development of fusigenic liposome vectors have been crucial to the progress of effective gene therapy strategies directed at renal structures in animal models. Many investigations have provided experimental models for gene delivery systems but the most efficient renal gene transfer was obtained from intrarenal injection or perfusion of explanted kidneys in transplantation. Continued technologic advances in vector systems and promising results in human and animal gene transfer studies make the use of gene therapy an encouraging strategy. Cell therapy, a tool for gene therapy, is based on the ability to expand specific cells in tissue culture to perform differentiated tasks and to introduce these cells into the patient either in extracorporeal circuits or as implants as drug delivery vehicles of a single protein or to provide physiological functions. These new approaches may result in therapeutic modalities that diminish the degree of renal failure and the time needed to recover renal function in acute tubular necrosis. This article specifically examines the present prospects of gene developing therapies in the treatment of ARF.
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