Gene Therapy 101: Understanding the Basics of Gene Therapy

The body is made of genes that are stored in the chromosomes. The chromosomes in the cell nuclei are made up of DNA. The work of DNA is to store information that determines unique traits. Genes form a part of DNA, which controls different characteristics like hair and eye color, height, and other attributes. Every person has two copies of a gene from the biological parents. In short, genes provide information on making proteins. Unfortunately, genes can vary, known as gene mutation. The slight change in the DNA within the genes alters protein digestion. The modification can be inherited, come with age, or from environmental elements like radiation and chemicals. That is where gene therapy comes in. This article will explore more on gene therapy and its basics.

How Gene Therapy Works

Gene therapy aims at altering genetic codes to recover critical proteins. Remember, proteins are fundamental in cells and the structural base of the tissues. Therefore, gene mutation impacts protein production and function. So, fixing or compensating it is essential to discover the critical roles of these proteins so the body can function correctly. 

This method uses genetic materials to prevent or treat disease. The DNA and RNA delivered have instructions as to the production of the protein. It compensates for the alterations in various ways. First, a new genetic material is introduced in the cells. If the altered gene is causing a fault in a protein, therapy helps transfer a normal gene to recover the critical function of the protein. Genome editing is another method used in gene therapy. Instead of adding a new material, this approach introduces gene-editing tools that change the cell. It becomes more accessible and more effective to read all the DNA in a plasmid through a sequencing method like whole plasmid sequencing. It eliminates the need to use a primer, and sequencing is faster.

The gene-editing tools and genetic material introduced in the cell don’t function. Instead, a vector is utilized to deliver the material. A vector is like a package for providing a specific message. For instance, viruses make significant vectors because they are great at entering cells. Researchers have also learned how to remove viruses to prevent and treat diseases. 

The Potentials of Gene Therapy

Gene therapy has a promising future. Although it still faces challenges, there is much to look forward to. The potentials include;

Treat Fatal Disease. Gene therapy can help address diseases that have limited treatment options. Without treatment options, many inherited disorders may end up in severe disability or death. That is where gene therapy is helping slow down or completely stop the disease.

Target Cause. Gene therapy is helping researchers design various kinds of treatments. These treatments can target numerous genes in the body. 

Early Treatment. If gene therapy is introduced early after diagnosis, there is a higher chance of stopping or reversing the damage. 

Key Takeaways

Generally, gene therapy is a scientific approach that helps treat or prevent disease by correcting the underlying genetic issue. The method lets doctors and scientists treat any disorder by altering the genetic makeup instead of surgery or drugs. So, it helps fix a disorder, fight disease, remove a dysfunctional gene, and remove DNA.