Genes within cells are critical to your health. If a gene is defective, it can make you sick. As medicine advances, researchers and medical professionals search for a way to treat defective genes, either by modifying them or replacing them. According to the Mayo Clinic, gene therapy is an experimental technique that replaces a faulty gene or adds a new, healthy gene to cure diseases or help your body fight diseases.
Gene therapy has multiple applications, including:
Replacing mutated genes: Sometimes genes stop working correctly or don’t work at all. This can cause diseases that are difficult to fight, such as cancer.
Fixing mutated genes: Mutated genes can create diseases, or make it more difficult for your body to fight disease.
Making diseased cells easier to find in the immune system: Sometimes, your body can’t find an illness because your immune system doesn’t recognize it. In the future, gene therapy may help you train your immune system to recognize dangerous cells.
Currently, gene therapy isn’t available as a medical procedure and is only available through clinical trials. As of 2017, The U.S. Food and Drug Administration has approved three forms of gene therapy products. Two of these products reprogram a patient’s existing cells to attack deadly cancer, and recently, the newest product targets a form of an inherited vision loss caused by mutation of a certain gene. Gene therapy will be beneficial in aiding in the treatment of cystic fibrosis, heart disease, diabetes, hemophilia, AIDS, cancer, and much more
Function of Cells and Genes
The FDA defines cells as the basic building blocks of living things. Human bodies are filled with trillions of cells, and within each cell, are thousands of genes. These genes provide the information needed to create muscles, bones, features, and more. Genes aid in the function of digestion, creating energy and physical growth.
Genes also contain our DNA, which controls how our body forms. When a gene doesn’t work properly, our bodies might experience deformity or disease. Gene therapy attempts to address the genes that aren’t working properly.
How Does Gene Therapy Work?
Sometimes a gene can be defective or missing at birth, or a gene can mutate in adulthood, causing health problems and diseases. Throughout the many clinical trials and research conducted on gene therapy, researchers have found that successful gene-based therapies rely on three elements, including:
A well-defined disease gene
A therapeutic gene
An efficient gene delivery system, known as a vector
Gene therapy seems to be specifically successful in dermatology. As the largest organ on the human body, skin is easily accessible for gene delivery. It’s also easily manipulated and evaluated, making it a primary target for gene therapy. Having an efficient vector is required for successful gene therapy. These vectors are genetically engineered to deliver the gene to the host. Viruses are particularly good at delivering genetic materials into a cell, and can therefore be used as successful vectors.
Gene Transfer Techniques
The vector will be injected either inside the body or outside the body, depending on which part of the body needs the gene therapy. The vector will contain the desired, healthy gene and is introduced to the cells.
There are two techniques for gene transfer, including in vivo gene therapy and ex-vivo gene therapy:
In vivo gene therapy: Genetic material is transferred using a vector that is directly injected into the patient
Ex vivo gene therapy: Cells are collected from the patient, genetically manipulated, and then administered to the patient
As we previously stated, viruses are particularly successful acting as vectors. Viruses are able to recognize specific cells and carry genetic material to the cell’s genes. Before the virus vector is administered to the patient, researchers will remove the disease-causing gene from the virus, and replace it with the desired genes required to stop the disease. Non-viral methods are possible and can be applied through electrical field-mediated gene transfer, magneto-permeabilization, and micro-needles.
Risks
Due to the method of utilizing viruses as a vector, there can be possible risks that it’s important to be aware of.
The viral genetic application can present the following risks:
Unwanted immune system reaction
Targeting the wrong cells
Infection caused by the virus
Possibility of causing a tumor
Our biorespository services offer tissues and cells from multiple collection samples, including melanoma, stomach tissue, lung tissue, kidney tissue, prostate tissue, and more. If you have any questions, contact our team.
Resources:
https://premier-research.com/bylined-articles/gene-therapy-in-dermatology-transfer-techniques-and-delivery-systems/
https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619
https://premier-research.com/the-state-of-gene-therapy-in-dermatology/
https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss
https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work
