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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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Updated: Mar 11, 2026

A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice

A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice

Published on: April 16, 2018

Cardiac Gene Delivery Using Recirculating Devices.

Melissa J Byrne¹, David M Kaye²

¹Heart Failure Research Group, Baker IDI Heart and Diabetes Institute, 75 Commercial Rd, Melbourne, VIC, 3004, Australia. melissa.byrne@bakeridi.edu.au.

Methods in Molecular Biology (Clifton, N.J.)

|December 3, 2016

Summary

This summary is machine-generated.

This study presents a novel percutaneous recirculation method for effective cardiac gene delivery. This technique aims to overcome challenges in translating gene therapy for heart failure (HF) into clinical practice.

Keywords:

Cardiac Gene delivery Percutaneous Recirculation Targeted

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Area of Science:

Cardiovascular Research
Molecular Medicine
Gene Therapy

Background:

Heart failure (HF) remains a significant clinical challenge with limited treatment options for advanced cases.
Understanding the molecular pathogenesis of HF has identified novel therapeutic targets.
Gene therapy holds promise for treating HF, but clinical translation is hindered by delivery challenges.

Purpose of the Study:

To describe a novel percutaneous recirculation method for cardiac gene delivery.
To address the need for large-scale, targeted gene delivery in the heart.
To facilitate the clinical translation of gene therapy for heart failure.

Main Methods:

Development and description of a percutaneous recirculation technique.
Application of the method for delivering therapeutic agents, including genes, to the heart.
Focus on a minimally invasive approach for cardiac intervention.

Main Results:

Successful delivery of potential therapeutic agents, including genes, to the heart was achieved.
The described method offers a viable approach for targeted cardiac gene delivery.
Demonstrated feasibility of the percutaneous recirculation technique in preclinical settings.

Conclusions:

The percutaneous recirculation method represents a promising advancement for cardiac gene therapy.
This technique may overcome key hurdles in translating gene therapy for heart failure to the clinic.
Further research and clinical trials are warranted to validate this delivery approach.