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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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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

Percutaneous approaches for efficient cardiac gene delivery.

Kiyotake Ishikawa¹, Jaume Aguero, Charbel Naim

¹Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, 1470 Madison Avenue, New York, NY 10029-6574, USA. kiyotake.ishikawa@mssm.edu

Journal of Cardiovascular Translational Research

|June 11, 2013

Summary

Gene therapy shows promise for heart failure, but challenges in delivery and efficiency remain. This review highlights less invasive percutaneous and endovascular injection methods for cardiac gene therapy, focusing on safety and efficacy.

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Area of Science:

Cardiology
Molecular Biology
Biomedical Engineering

Background:

Gene therapy is under development for heart failure treatment.
Current gene therapy approaches face challenges in vector efficiency and delivery methods.
Translating cardiac gene therapy to clinical practice requires collaboration between biologists and cardiologists.

Purpose of the Study:

To review percutaneous and endovascular gene delivery methods for cardiac applications.
To summarize features of currently available percutaneous gene delivery techniques.
To address the need for less invasive approaches in advanced heart failure patients.

Main Methods:

Focus on injection-based gene delivery techniques.
Emphasis on percutaneous and endovascular approaches.
Summarization of existing percutaneous gene delivery methods and their characteristics.

Main Results:

Gene transduction efficiency is often linked to invasiveness.
Less invasive methods are preferred for patients with advanced heart failure.
Percutaneous and endovascular approaches offer potential for improved cardiac gene therapy delivery.

Conclusions:

Optimizing gene delivery methods is crucial for clinical translation of cardiac gene therapy.
Percutaneous and endovascular techniques represent promising avenues for safer and more effective gene delivery.
Further research into these less invasive methods is essential for advancing heart failure treatment.