Gene Therapy

Introduction

Gene therapy is the process of modifying the genes contained within your body’s cells in an attempt to treat or prevent disease.

Genes contain your DNA, which is the genetic code that regulates many aspects of your body’s structure and function, from making you grow taller to managing your body’s internal organs. Genes that do not function properly can result in disease.

Gene therapy is still under investigation by researchers as to how and when it should be used. Gene therapy is now only offered in the United States as part of a clinical trial, which is not widely available elsewhere.

Gene Therapy

Gene therapy is a technique for correcting genetic defects in order to cure an illness or help your body fight disease more effectively.

Researchers are looking into a variety of approaches to accomplish this, including:

Replacing genes that have been mutated. Disease develops in some cells as a result of particular genes malfunctioning or ceasing to function altogether. It is possible that replacing the faulty genes will aid in the treatment of some disorders. For example, a gene known as p53 is generally responsible for preventing tumour growth. A number of different forms of cancer have been related to mutations in the p53 gene. If doctors were able to successfully repair the faulty p53 gene, it is possible that cancer cells would be killed.

Putting a stop to mutated genes. Disease-causing genes might be switched off so that they no longer promote disease, and similarly beneficial genes that prevent disease may be activated to prevent the disease from spreading further.

Increased visibility of diseased cells to the immune system is beneficial.. Because sick cells are not recognised as intruders by your immune system in some situations, your immune system does not evade them. The use of gene therapy could allow doctors to train your immune system to spot cells that pose a threat to your health.

Risks

There are certain hazards associated with gene therapy. A gene cannot be simply introduced into your cells because of the complexity of the process. Instead, it is normally conveyed through the use of a carrier, which is referred to as a vector.

In gene therapy, viruses are the most commonly used vectors because they are capable of recognising specific cells and delivering genetic material into the cells’ genes. The virus’s original illness-causing genes are removed by the researchers, who then replace them with ones that prevent disease from spreading.

The following hazards are associated with this technique:

Immune system reaction that was not desired. The newly imported viruses may be recognised as intruders by your body’s immune system, which may respond by attacking them. Organ failure may occur in severe situations as a result of the inflammation.

The wrong cells are being targeted. Considering that viruses can infect a variety of different types of cells, it is probable that the modified viruses will also infect non-targeted cells in addition to the targeted cells that have been transformed. If this occurs, healthy cells may be destroyed, which may result in the development of other illnesses or diseases, such as cancer.

The virus is the source of the infection. Following their introduction into the body, it is possible that viruses regain their potential to cause disease that they originally possessed.

There is a chance of producing a tumour. The insertion of additional genes in the wrong location in your DNA has the potential to result in the growth of tumours in some cases.

The Food and Drug Administration and the National Institutes of Health are actively monitoring the gene therapy clinical trials currently ongoing in the United States to ensure that patient safety concerns are given high consideration during the research process.

Expectations When You Visit

At this time, the only method for you to obtain gene therapy is to take part in a clinical trial, which costs money. Generally speaking, clinical trials are research studies that are used to assess whether a gene therapy strategy is safe for patients. They also aid clinicians in their understanding of the consequences of gene therapy on the human body and mind.

The particular operation you will have will be determined by the ailment you have and the type of gene therapy that is being utilised.

For example, with one sort of gene therapy, the following is true:

It is possible that you will have blood extracted or that bone marrow will be harvested from your hip bone with a big needle.

Then, in a laboratory setting, cells derived from the blood or bone marrow are exposed to a virus or another sort of vector that contains the genetic material that is sought.

The cells from the lab are then injected into your body by a vein or tissue, where your cells pick up the vector and the changed genes that were introduced into them in the laboratory.

A virus is not the only vector that can be used to introduce changed genes into the cells of your body. Other vectors can be used to do the same thing. Other vectors that are currently being investigated in clinical studies include:

Stem cells are a type of cell that can regenerate itself. Stem cells are the cells that are responsible for the formation of all other cells in your body. In the case of gene therapy, stem cells can be educated in a laboratory to become cells that can aid in the treatment of disease.

Liposomes. These fatty particles have the ability to transport the new, therapeutic genes to the target cells and integrate the genes into the DNA of the cells in which they are transported.

Conclusion

The findings of such trials suggest that gene therapy has the potential to cure a wide range of genetic diseases, and that the procedures appear to pose minimal risks to the patients. However, the efficiency of gene transfer and the expression of corrective genes in human patients are still very low, as evidenced by the results of these trials.