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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 inserted. The...

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Related Experiment Video

Updated: May 24, 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

Gene delivery technologies for cardiac applications.

M G Katz¹, A S Fargnoli, L A Pritchette

¹Department of Thoracic and Cardiovascular Surgery, Sanger Heart and Vascular Institute, Cannon Research Center, Carolinas HealthCare System, Charlotte, NC, USA.

|March 16, 2012

Summary

Gene therapy offers new hope for ischemic heart disease (IHD) and heart failure (HF) by improving genetic transfer methods. Research focuses on targeted delivery for IHD and global cardiomyocyte transduction for HF, enhancing treatment efficacy.

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

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Gene Transfer for Ischemic Heart Failure in a Preclinical Model

Gene Transfer for Ischemic Heart Failure in a Preclinical Model

Published on: May 15, 2011

Area of Science:

Cardiovascular Medicine
Molecular Biology
Gene Therapy

Background:

Ischemic heart disease (IHD) and heart failure (HF) are leading causes of death.
Advances in molecular pathology and vector technology enable gene-based therapies for cardiac conditions.
Effective gene transfer is crucial for therapeutic success.

Purpose of the Study:

To review gene-transfer strategies for coronary artery disease and heart failure.
To discuss advancements in direct and transvascular gene delivery methods.
To evaluate the success of vector-mediated cardiac gene delivery.

Main Methods:

Review of current gene therapy research in models of IHD and HF.
Analysis of localized gene delivery via catheters and stents for IHD.
Examination of global cardiomyocyte transduction for HF treatment.

Main Results:

Gene therapy strategies are evolving for IHD and HF.
Localized delivery targets angiogenesis and endothelial function in IHD.
Global transduction aims to inhibit apoptosis, reduce remodeling, and increase contractility in HF.

Conclusions:

Gene therapy holds significant potential for treating IHD and HF.
Optimizing gene transfer efficiency is key to clinical success.
Targeted approaches differ between IHD and HF, reflecting disease-specific needs.