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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

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Related Experiment Video

Updated: Apr 1, 2026

Hydrodynamic Renal Pelvis Injection for Non-viral Expression of Proteins in the Kidney

Hydrodynamic Renal Pelvis Injection for Non-viral Expression of Proteins in the Kidney

Published on: January 8, 2018

Kidney-specific Sonoporation-mediated Gene Transfer.

Ryo Ishida¹, Daisuke Kami², Tetsuro Kusaba¹

¹Division of Nephrology, Department of Internal Medicine, Graduate School of Medical Science Kyoto Prefectural University of Medicine, Kyoto Prefectural University of Medicine, Kyoto, Japan.

Molecular Therapy : the Journal of the American Society of Gene Therapy

|October 1, 2015

Summary

Local kidney sonoporation enables efficient delivery of systemically administered oligonucleotides to tubular epithelium. This technique shows promise for treating ischemic acute kidney injury by reducing tumor necrosis factor alpha expression.

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Last Updated: Apr 1, 2026

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Published on: January 8, 2018

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Area of Science:

Biomedical Engineering
Nephrology
Molecular Therapy

Background:

Sonoporation facilitates targeted agent delivery to organs via systemic administration, minimizing adverse effects.
Previous applications of sonoporation have involved diverse materials and organs.
Tumor necrosis factor alpha (TNFα) is a key mediator in renal ischemia-reperfusion injury.

Purpose of the Study:

To demonstrate efficient oligonucleotide transfer to the kidney's tubular epithelium using local sonoporation.
To investigate the potential of renal sonoporation for treating ischemic acute kidney injury.
To assess the efficacy of targeting TNFα with small interfering RNA (siRNA) in a murine model.

Main Methods:

Local sonoporation applied to the kidney.
Systemic administration of oligonucleotides.
Ultrasonic wave irradiation to induce microbubble collapse and transiently alter glomerular permeability.
Targeting TNFα using siRNA in a murine model of renal ischemia-reperfusion injury.

Main Results:

Highly efficient and specific transfer of systemically administered oligonucleotides to the tubular epithelium was achieved.
Ultrasonic wave irradiation transiently increased glomerular permeability, allowing oligonucleotide passage.
Reduction in TNFα expression via single gene transfer significantly inhibited kidney injury markers.

Conclusions:

Local sonoporation to the kidney offers a highly efficient method for delivering systemically administered oligonucleotides to tubular epithelial cells.
This approach holds potential for clinical applications in treating ischemic acute kidney injury.
Systemic siRNA administration combined with temporary, local sonoporation is a promising strategy for gene therapy in kidney injury.