Gene Therapy Basics – 4 Things You Need To Know

Updated on June 7, 2021

Gene therapy has been studied for around 40 years and is a relatively new, revolutionary type of medical treatment. Companies like Thermogenesis are making cell and gene-based therapy a potential reality. This article will briefly cover the basics of what gene therapy is and how it works.

Overview Of Genes

Made of DNA, genes are a blueprint of our body and the basic unit of heredity. Humans have a whopping 20,000 – 25,000 genes in their body and typically get two copies of DNA from each parent.

What Is Gene Therapy?

You may be wondering, what is gene therapy? Gene therapy introduces, removes, or changes genetic material into a patient’s cells to treat a specific disease. 

Gene changes are also called mutations. When a gene changes in a way that causes disease or disorder, gene therapy may help. The genetic material that is transferred changes how a protein or (group of proteins) is produced by the cell.

This new genetic material or working gene is delivered into the cell by using a vector. Vectors are carriers designed to provide therapeutic genetic material directly into a cell. Usually, viruses are used as vectors because they have become very good at creeping up into and infecting cells. But in this instance, their purpose is to insert the new genes into the cell. Some types of viruses being used are typically not known to cause disease, and other times the viral genes known to cause disease are removed. No matter what the type, all viral vectors are tested multiple times for safety before being used. Either the vector can be delivered outside the body (ex-vivo treatment), or the vectors can be injected into the body (in-vivo treatment).

What Classifies As A Rare Disease?

Gene therapy can possibly help treat rare diseases in patients. The National Institutes of Health define rare diseases as “any disease or disorder affecting fewer than 200,000 people in the U.S.”. There are around 7,000 rare diseases at the moment, affecting a total of approximately one in ten people. A lot of these rare diseases are caused by a genetic mutation inherited from one or both parents.

Which Diseases Can Gene Therapy Potentially Help?

Forty-five percent of gene therapies up for approval over the next five years are predicted to focus on cancer treatments. Thirty- eight percent are expected to treat rare inherited genetic disorders. Additionally, gene therapy can help add to and change genes that are non-functioning – creating an extraordinary opportunity to assist with rare inherited disorders possibly passed along from parents. The vast majority of gene therapies are currently being studied in clinical trials.

Some of the inherited diseases include but are not limited to:

  • Hematology/blood disorders like sickle cell disease
  • Neurological disorders that affect the brain and spinal cord
  • Musculoskeletal diseases
  • Retinal disorders

Overall, gene therapy is an interesting and promising new concept, but it is still under research. If you want to learn more, check out sites like Thermogenesis for information about the latest cell and gene-based technologies.

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