Connect with us


The Role of Gene Therapy in the Treatment of Genetic Disorders & Other Diseases



Gene therapy is supposed to have contributed to significant medical advances within two decades. It has progressed from the initial conceptual stage to the development of technology and lab research to certain clinical translational trials associated with a wide range of deadly diseases. Here are some of the genetic disorders or diseases that could be treated with gene therapy.

Severe Combined Immune Deficiency or SCID

Severe Combined Immune Deficiency is supposed to be a potentially terminal primary immunodeficiency that is marked by the combined absence of B-lymphocyte and T-lymphocyte function. There are minimum 13 different types of genetic defects that could cause SCID. All these defects would be culminating in extreme susceptibility and almost no resistance to some very serious infections. SCID is usually regarded as the most severe or deadly among the primary immunodeficiency. However, today, fortunately certain effective treatments, like stem cell transplantation, has been introduced and implemented for curing the disorder. The future seems quite promising as gene therapy is making ground-breaking progress.

Chronic Granulomatous Disorder or CGD

The chronic granulomatous disease is supposed to be a genetic or inherited immune system disorder which would be occurring when a kind of white blood cell called phagocyte that generally aids your body combat infections actually does not function properly. As such, the phagocytes are unable to safeguard your body from certain fungal and bacterial infections.

Patients suffering from the chronic granulomatous disease often would be developing pneumonia, skin infections, lung infections, liver infections, lymph node infections, gastrointestinal inflammation or some other infections. Stem cell transplantation could be providing a perfect cure for CGD. However, the decision for treatment with stem cell transplantation would be depending on a variety of factors including personal preference, prognosis, and donor availability. Gene therapy is presently being explored for an effective CGD treatment. In Germany, two patients suffering from CGD were treated by reconstituting immune systems using an effective gene therapy. The patients with reconstituted immune systems enjoy complete protection from microbial infections for a minimum of two years. Browse for more information.


Patients born with Hemophilia are incapable of inducing blood clots and generally suffer from both internal and external bleeding that could prove to be fatal. A clinical study was conducted in the U.S.A. recently where the therapeutic gene actually was infused into patient’s liver. The patient could then experience normal blood clotting. However, the therapeutic effect was short-lived as the genetically modified liver cells were identified as foreign and then rejected by the patient’s healthy immune system. This is supposed to be the same issue associated with organ transplants. Curative results from gene therapy could be obtained by alternative gene delivery techniques and stratagem.


Gene Therapy promises treatment for acquired diseases in future. Multiple gene therapy techniques have already been developed for treating a broad spectrum of cancers including therapeutic gene vaccines, oncolytic virotherapy, suicide gene therapy, and anti-angiogenesis. Today, gene therapy trials for cancer are at advanced stage. Proper treatment for pancreas and prostate cancers seem promising with gene vaccine trials. Various clinical trials are in progress for the treatment of skin, brain, colon, liver, kidney, and breast cancers. Moreover, the recent advancement in gene therapy has resulted in innovative treatments for Huntington’s disease, Parkinson’s disease, and other such neurodegenerative diseases.

Click to comment

Leave a Reply

Your email address will not be published. Required fields are marked *