Children suffering with rare life threatening, inherited immunodeficiency disorders can now be treated with the investigational gene therapy which proves to be efficient in restoring the immunity. This research was done in collaboration with National Institutes of Health.
It was found that almost 50 children who received this gene therapy replenished with their immunity 2- 3 years later and did not require any further treatment for their condition know as ADA-SCID ( severe combined immunodeficiency due to adenosine deaminase deficiency ). These findings were later published on New England Journal of Medicine.
It is observed that 1 in 200,000 to 1,000,000 babies acquire SCID disease as new born. This happens due to mutation in the mutation in the ADA gene which is responsible for ceasing the activity of adenosine deaminase enzyme . This adenosine deaminase enzyme is required for maintaining a healthy immune system.
This disease is usually fatal within 2 – 3 years of birth if left untreated. This disease is generally associated with the immune system making the person susceptible to many diseases which eventually in fatal.
The director of NIH’s National Institute of Allergy and Infectious Diseases (NIAID) said that according to the research, gene therapy could possibly help in overcoming this disease in new born babies as well as in children. Also, this gene therapy is a one time treatment after which one person can live a healthy life with great immune system.
There are other ways also available to treat this disease but no treatments are effective as gene therapy. Other treatments includes, enzyme replacement therapy, Transplants of blood-forming stem cells etc. But these treatments are only effective for certain period of time and might require re transfusion within a short period of time.
The latest experiments suggest that the lentiviral gene therapy is the safest of all the existing gene therapy and has better results than others. This procedures involves inserting ADA gene into the patient’s own blood forming stem cells.
These are proven to be effective and is a one time therapy that would definitely save lives of millions of people.