Gene Therapy 101 with Dr. Kevin Nash

Quick Overview

Dr. Kevin Nash, an expert in gene therapy, explains the basics of gene therapy in a lecture. Gene therapy is an experimental technique that involves delivering genes into an organism, specifically into cells. The goal is to correct genetic problems by introducing genetic material that can replace or fix the faulty genes. One method of delivering genes is through viral vectors, which are modified viruses that can transport the genes into cells. Adeno-associated virus (AAV) is a promising viral vector because it is safe, does not cause disease or immune responses, and can deliver genes to neurons in the brain. Dr. Nash also discusses different approaches to gene therapy, such as gene replacement therapy and protein replacement therapy, as well as new methods of targeting the brain without direct injections. The duration of gene therapy’s effects can vary, but studies have shown long-term protein production for at least 6 to 10 years.


In this talk, we will explore the field of gene therapy with Dr. Kevin Nash, an Assistant Professor in the Department of Molecular Pharmacology and Physiology at the University of South Florida. Dr. Nash is a member of the FIRE Consortium and has a strong interest in gene therapy, particularly in relation to Angelman Syndrome. He will discuss the basics of gene therapy, its potential applications, and the challenges involved in its implementation.

What is Gene Therapy?

Gene therapy is an experimental technique that involves delivering genes into an organism, specifically targeting certain cells within the body. The goal is to introduce genetic material that can correct a problem caused by a lack of a specific gene. For example, in the case of Angelman Syndrome, the UBE3A gene is missing, so the aim is to reintroduce this gene into the affected cells.

Understanding DNA and Genes

DNA is the stored information that encodes for everything in our bodies. It acts as an instruction manual for cells to produce proteins. Each gene in our DNA encodes for a specific protein. If there is a problem with a gene, it can lead to a problem with the corresponding protein. In gene therapy, we isolate the correct gene and introduce it into the cells to restore normal function.

Methods of Gene Delivery

There are various methods of delivering genes into cells. One approach is to simply place the DNA on the cell and hope it enters. However, this method is not very effective. Other methods include physical methods like electroporation, which uses a small electrical charge to open up the cell and allow the DNA to enter. Chemical methods involve covering the DNA with molecules that can transport it into the cell. The most promising method for humans is the use of viral vectors.

Viral Vectors in Gene Therapy

Viral vectors are modified viruses that can deliver genes into cells without causing disease. One of the most promising viral vectors is adeno-associated virus (AAV). AAV is safe, does not cause an immune response, and can be modified to remove all viral genes. It has been extensively studied in clinical trials and has shown long-term protein production. AAV can also target neurons in the brain, making it suitable for gene therapy in neurological disorders.

Challenges and Future Directions

One of the challenges in gene therapy is finding the best way to deliver genes to neurons and target as many neurons as possible. Researchers are exploring different techniques, including gene replacement therapy and protein replacement therapy. They are also investigating alternative methods of delivery, such as injecting the virus into the bloodstream, which could be less invasive for patients. Additionally, researchers are working on neuron-specific promoters to ensure gene expression occurs only in the desired cells.


Gene therapy holds great promise for treating genetic disorders by delivering genes into cells to correct underlying problems. Dr. Kevin Nash has provided an overview of gene therapy, its applications, and the challenges involved. With ongoing research and advancements in viral vectors, gene therapy has the potential to revolutionize the treatment of various diseases.

Talk details

  • Title: Gene Therapy 101 with Dr. Kevin Nash
  • Author(s): Kevin Nash
  • Author(s)’ affiliation: University of South Florida
  • Publication date: 2016-12-02
  • Collection: 2016 FAST Science Summit