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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...

What is Genetic Engineering?

What is Genetic Engineering?

iPS Cell Differentiation

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.

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Related Experiment Video

Updated: May 14, 2025

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons

Published on: January 7, 2019

Developing a gene therapy for Charcot-Marie-Tooth disease: progress and challenges.

Marina Stavrou¹, Alexia Kagiava¹, Irene Sargiannidou¹

¹Neuroscience Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.

Regenerative Medicine

|April 12, 2025

Summary

Gene therapy offers new hope for Charcot-Marie-Tooth (CMT) disease, a progressive neuropathy. While promising, challenges in delivery, safety, and scalability must be overcome for clinical readiness.

Keywords:

Charcot-Marie-Tooth disease clinical trial readiness gene therapy inherited neuropathy preclinical testing

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Published on: June 15, 2018

Area of Science:

Neurology
Genetics
Biotechnology

Background:

Charcot-Marie-Tooth (CMT) disease is a group of inherited, progressive neuropathies.
Current management for CMT is limited to symptomatic treatments.
No approved therapies specifically target the underlying causes of CMT.

Purpose of the Study:

To review recent advances in gene therapy for CMT.
To highlight progress in gene silencing, replacement, and editing strategies.
To summarize preclinical successes and identify translational challenges.

Main Methods:

Review of current literature on gene therapy approaches for CMT.
Analysis of preclinical data for gene silencing, replacement, and editing.
Evaluation of delivery technologies like AAV vectors and nanoparticles.

Main Results:

Gene therapy strategies show promise for various CMT types.
AAV vectors and nanoparticle systems are leading delivery technologies.
Significant challenges remain, including blood-nerve/brain barrier penetration, immunogenicity, toxicity, and scalability.

Conclusions:

Optimizing therapeutic delivery is crucial for clinical readiness.
Enhancing safety profiles and developing monitoring biomarkers are essential.
Advancing CMT treatments requires addressing these translational hurdles.