What is Gene Therapy

Gene therapy is the way by which not only genetic disorders but also several other disorders are being cured and treated. The development of the health departments has led to this massive discovery of treatment. In order to under the procedure, let us take a short look through the following terms:

• Chromosomes: are threadlike structures of proteins and single DNA molecules, carrying genomic information from one cell to another.
• Gene: the basic unit of inheritance, gene is passed from the parents to the offspring, containing several biological and characteristic traits as well as information.
• Genome: It is the entire set of DNA instructions. In the nucleus of the human body’s cells, there are a total of 23 pairs of genomes.
• Inheritance: a trait or a variant that is encoded in DNA and passed from parents to offspring.
• Genetic code: refers to the instructions contained in a gene that direct the cell to make specific proteins.

The origin of gene therapy dates back to 1970, when it was not well accepted. The whole concept of replacing or fixing a faulty gene, which can lead to the cure of the disease, has been in wide use nowadays. It is believed that several genetic disorders, such as cystic fibrosis, beta-thalassemia, and hemophilia, can be cured in the near future using gene therapy. It is considered one of the greatest discoveries of the time, as diseases that feared people back in time like HIV infections are gradually seeing a scope to be cured utilizing this method.

Even though the research is at an early stage, researchers are well aware of the methodology. The faulty gene is replaced or fixed either inside the patient’s body or the gene undergoes fixing outside their body and is then inserted into the patient’s body. Additionally, viral replication is used. The disabled virus carries the therapeutic gene, which it uses to infect the target cell directly and further integrate the correct gene into the target cell’s genome. This genome serves as the template for the correct synthesis of proteins. The protein synthesis is temporary or permanent.

The potential risks of this modern therapy include the following:                                     

• Cancer
• Allergies
•  Damage of organs and tissues

SCID treatment: The initial application and succession of gene therapy

The succession of this process dates back to 1990, when it was first applied to 4-year Ashanthi de Silva, who was born with SCID (severe combined immunodeficiency) disorder that made her immune system incompetent to fight infections. This was primarily due to the death of her T-cells, because of the absence of ADA (adenosine deaminase). Back then, a healthy ADA gene was introduced into her blood cells through a disabled virus that could not spread in her body.

Vector adjustments to enhance the application mode:

There are two kinds of vectors that are used, while the

Gene therapy in the treatment of cancer:

It is common knowledge that mutations or genetics can cause cancer, which has been linked to multiple fatalities in recent years. People were initially unaware of the cause, and later on, they were unaware of the remedy. We now have hope and believe that it is a disease that can be cured in a number of ways, thanks to diligent research and hard work. Of all the ways of treating it, gene therapy has proven to be extremely beneficial. 

• It is possible to swap out the dysfunctional genes for working ones. 
• restricting or outlawing these oncogenes. 
• Promoting fluency in processes like chemotherapy through the insertion of new genes into the cancerous gene. 
• Using techniques to prevent angiogenesis in cancer patients(anti-angiogenesis). 

These ways have worked out well in order to promote the recovery of the patients from these diseases. However, these methods are not functional during the last stage of the disease.

A recent instance of gene therapy

Experts and researchers from Fuhan University have diagnosed six children with the genetic condition of deafness. It is an autosomal recessive disorder that was previously considered incurable, and people tended to remain unchecked or undiagnosed. Scientists discovered the treatment for this hereditary illness in Shanghai on January 29. They used an adeno-associated virus to mount the OTOF transgene in order to introduce the gene drug RRG-003. The outcome of this operation was successful, as the six children reportedly gained back their hearing capabilities and their speech as well.

In the long run, how beneficial is gene therapy?

Since this idea is still relatively new in the field of medical science, it is somewhat unknown how this method will affect things on a larger or longer scale. There are several factors that may influence the after-effects of this process, one of which is how and where the therapy has been introduced into the patient’s body. After the initial introduction of the new cells into the body of the patient, the immune system might take time to adjust, and thus the body might exhibit symptoms such as fever, fluctuations in blood pressure, rigors, et cetera. There are chances that the introduction of new cells might cause the generation of new cancerous genes.
However, the advancement of the whole method has led to safer results, and in the future, it will be the greatest cure for several life-threatening diseases.

Frequently Asked Questions:

1. What is gene therapy?

Gene therapy is a modern technique in which new and normal genes are used to replace defective or damaged genes. This technique has become excessively crucial in the medical field at a global level. 

2. Is gene therapy a cure?

Gene therapy has been the top reason for the improvement of several gene-related disorders, gene disorders, and many others. Thereafter, this method can be called a cure. 

3. Is gene therapy very expensive?

As this method has begun progressing in very recent times and a very few of them actually know about it, this can be expensive. It ranges somewhere between $1 million and $2 million.