Gene therapy

Overview:

The body is made up of genes that contain DNA. The DNA stores information to determine the specific traits of a person and is responsible for how the body of the person shapes up. It determines a person’s skin and hair color, height, way of walking, and breathing, and even how their gut functions.

Genes are responsible for producing proteins in the body which play a very vital role in all the functions. Any issue with the genes can change how the proteins work and are called gene variants. Hence when there is a problem with the genes, it causes disorders and diseases.

What are Gene Variants:

Variations in the genes occur due to exposure to certain chemicals or because of environmental changes. Cells usually identify these variations or changes and repair them. However, at times the cells cannot be repaired and cause diseases or health conditions where treatment is needed. The intriguing part is that the untreated gene variants can be passed on to future generations from biological parents leading to disorders at an early age.

Gene therapy addresses the underlying reason for the disease and helps in fixing the missing parts or overlooked errors. The treatment helps in fixing a malfunctioning gene and if that is not possible it replaces that gene with a healthy one to cure the disease or gear up the body to fight the disease effectively.

The therapy has been approved by the U.S. Food and Drug Administration department for multiple health conditions like cancer, spinal muscular atrophy, hemophilia, and sickle cell disease. The therapy has also proved very successful in treating cystic fibrosis, heart ailments, diabetes, and aids.

What Are The Reasons Why Gene Therapy Is Done:

There are three primary reasons why gene therapy is carried out.

Correcting the genes: The therapy aims to switch off faulty genes that lead to health conditions so that they don’t progress the disease further. Simultaneously the healthy genes that help prevent health conditions are activated.
Replacing genes: Certain cells cause diseases because they don’t work optimally or don’t function at all. The therapy helps to replace these genes with healthy ones to stop the progression of certain diseases.
Making the immune system aware of the disease-carrying cells: Sometimes the immune system fails to target the diseased cells because it does not consider them to be a threat. The therapy trains the immune system to identify the cells as intruders so that they can be destroyed.

The Different Types OF Gene Therapy Products:

Gene therapy products are biological products that are regulated and approved by the FDA’s Center for Biologics Evaluation and Research (CBER). They include:

Plasmid DNA: These are round-shaped DNA molecules that are genetically modified to carry therapeutic genes into the cells of the human body.

Viral Vectors: Viruses are able to carry genetic material into the cells and hence some times gene therapy uses viruses that act as vectors to carry therapeutic genes in the human body and remove the cause of infectious diseases.

Bacterial Vectors: Similarly some bacteria can be modified and used as vessels to carry healthy and therapeutic genes to prevent certain diseases.

Gene editing: Gene therapy involves gene editing to disturb harmful genes and repair the mutated ones.

Patient-derived cellular gene therapy products: Cells are removed from the patient’s body, genetically corrected, and returned to the body.

Stem cells: All cells in your body are made from stem cells. These stem cells can be changed in a laboratory to enable them to fight diseases.

Liposomes: Liposomes help to carry modified therapeutic genes to the diseased cells and pass the genes into the DNA of the cells.

Procedure Details of Gene Therapy:

The procedure depends upon the type of gene therapy used.
Blood is taken out or the person’s bone marrow is extracted from the hip bone with the help of a large needle.
The cells from the blood or the bone marrow are subjected to exposure to a virus/ bacteria in a laboratory that has the required genetic material.
Once the vector has been given entry into the cells, the modified cells are injected back into the body of the person through a vein or tissue. The person’s body accepts the vector along with the modified cells.
Sometimes the vector is infused into the blood or a specific organ.

The Benefits Of Gene Therapy:

The therapy offers new hope for health conditions that presently have no treatment options and can cause disabilities or even death of the person. The therapy has proved in clinical trials to either stop or slow down the progression of these diseases.
Timely gene therapy can prevent further damage due to a disease in a person’s body. It has also proved to reverse the damage that has already happened.
Gene therapy identifies the cause of the disease to formulate treatments that work on specific genes in the body.

Risks Involved In Gene Therapy:

The modified gene cannot be easily delivered to the diseased or faulty cell. It must be carried with the help of a vector.
The body’s immune system may get threatened by the entry of the new vector which are usually viruses or bacteria and attack them. This leads to a reaction in the body that causes swelling or failure of organs.
The vectors have the chances of affecting more than one cell and damaging the healthy cells.
Once the vectors enter the body, there are chances of them causing infection in the person’s body leading to diseases.
There are chances of the therapy causing errors in the genes which may be the cause of cancer.
All the gene therapies may not work. There are chances of failure or the occurrence of side effects.

Conclusion:

With gene therapy, it is still the beginning. There are multiple challenges yet to be overcome to establish the treatment as an option to offer a cure for many diseases. The person choosing the treatment must be aware that the process is very long and tedious and has to undergo FDA approval. However, there is continuous research being carried out in the field and hopefully, in the future, the therapy will be able to prevent and reverse many rare and complicated conditions.

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