March 5, 2019
By: Pathsos team

Genetic Engineering in Medicine


An estimate states that there are around 37.2 trillion cells in our body. That’s around 37.2 trillion gene structures we could correct or replace with newer healthier genes to cure diseases like cancer. As optimistic and mind-blowingly innovative as the idea sounds, the process is still at its experimental stage and even though replacing all the 37 trillion cells and their genes will not be necessary, correcting just a few defective genes is a tricky business. 

What is Gene Therapy?

Gene therapy is a technique where the defective mutated genes inside living cells are replaced with healthier genes in order to treat and prevent diseases that are formed due to genetic mutation, for example, Cancer. Most of the diseases that gene therapy is used for has no definitive cure and hence this risky technique is in demand as a newer and more complicated disease spring up day-by-day.

It is a therapeutic process which aims to alter the genetic make-up of the person in the process of getting rid of the mutated genes.  This technique is aimed to fix genetic problems at the base and source. With advancements in genetic engineering and in over-all medical developments in the late 1990s, this technique grew popular among researches and clinical trials and research took off at full speed. Its types include somatic, which is not inheritable and germ-line, the inheritable kind.

The ways in which gene therapy works is an interesting topic. Under gene augmentation therapy, a functioning gene is put in the place of a lost gene; gene inhibition therapy is where faulty genes are blocked from expressing their working and the new genes are introduced in cells and in specific killing therapy, faulty genes are killed by sending a suicide gene and this results in the death of the cell.

What kind of cancers does the treatment cure?

Gene therapy is often used to treat genetic disorders and defects like cancer, and diseases that travel one's bloodline through their heredity. Cystic fibrosis, hemophilia, heart disease, AIDS are a few complex diseases among other immunodeficiencies like severe combined immunodeficiency that gene therapy could fix.

Research and side effects:

The most recent research in gene therapy is in the protecting cells against HIV,  genome editing for Duchenne muscular dystrophy,  using single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome,  association of PXR and CAR Polymorphisms and Antituberculosis Drug-Induced Hepatotoxicity, development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome to name a few. The most active research has been in cystic fibrosis and other severe immune deficiencies like adenosine deaminase (ADA) deficiency (a rare genetic disease that makes kids prone to serious infection), sickle cell anemia, thalassemia, hemophilia, and those with familial hypercholesterolemia.

 Side effects include toxic and inflammatory responses, in germ-line gene therapy, the development of fetus could be hindered due to the genetic changes made in the egg or the sperm level.  In ALS, amyotrophic lateral sclerosis, a progressive neurodegenerative disease,  the virus that is sent with the corrective gene could cause severe problems. In recent news,  children undergoing gene therapy had developed cancer in response to the treatment strategy. Vector-mediated insertional mutagenesis is a serious problem in children receiving SCID gene therapy.  In certain cases where gene therapy was used to treat thrombosis, gene transfer led to 70% increase in the thickness of inner layers of arteries when experimented on a rabbit using an enzyme called urokinase-type plasminogen activator, uPA.

Gene Therapy, being an expensive and experimental treatment for deadly diseases like cancer, seems to remain an experimental option but the situation promises to change as advancements in the medical and genetic engineering fields rise. People can avail gene therapy as of now by registering themselves into clinical trials and bear the massive cost that comes with it. But with a chance, cancer could be defeated on an expensive and critical road to their cancer cure.