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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 21, 2026

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

Methods to improve cardiac gene therapy expression.

Maria Cecilia Scimia¹, Kate E Sydnes, Daniel A Zuppo

¹Temple University, School of Medicine, Philadelphia, Pennsylvania, USA.

Expert Review of Cardiovascular Therapy

|October 24, 2014

Summary

This summary is machine-generated.

Gene therapy offers a promising treatment for heart failure, focusing on effective delivery methods to maximize cardiac cell transduction. Research explores overcoming viral barriers for improved gene expression in targeted tissues.

Keywords:

adeno-associated viruses antegrade injection cardiomyocytes clinical usage electroporation gene gun-mediated delivery gene therapy heart failure ischemia jet injection laser-based transfection magnetofection myocardial infarction retrograde injection sonoporation vascular delivery

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Last Updated: Apr 21, 2026

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

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model

Published on: June 11, 2020

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

Area of Science:

Cardiovascular Medicine
Molecular Therapy
Biotechnology

Background:

Gene therapy is an emerging strategy for treating heart failure.
Current clinical trials primarily use vascular access for gene delivery.
Developing optimal delivery methods is crucial for therapeutic success.

Purpose of the Study:

To review gene therapy delivery strategies for heart failure.
To identify barriers to viral vector transduction in cardiomyocytes.
To explore methods for enhancing gene expression in cardiac tissue.

Main Methods:

Review of current gene therapy research for heart failure.
Analysis of viral vector delivery systems.
Discussion of challenges in cardiomyocyte transduction and viral biodistribution.

Main Results:

Vascular access remains the primary delivery method, but alternative strategies are under development.
Successful gene therapy requires optimizing vector, gene, and delivery for high cardiomyocyte transduction.
Minimizing off-target viral spread and correlating dose to expression are key challenges.

Conclusions:

Effective gene therapy for heart failure necessitates overcoming biological barriers for efficient cardiac delivery.
Further research into novel delivery methods is essential to improve therapeutic outcomes.
Understanding viral transduction pathways is critical for advancing cardiac gene therapy.