Children with rare, inherited, life-threatening immunodeficiency disorders can now be treated with experimental gene therapy, which has been shown to be effective in restoring immunity. This research was carried out in collaboration with the National Institutes of Health.
It was found that almost 50 children who received this gene therapy restored their immunity 2-3 years later and did not need further treatment for their condition known as ADA-SCID (Combined Immune Deficiency). severe due to adenosine deaminase deficiency). These results were then published in the New England Journal of Medicine.
1 in 200,000 to 1,000,000 babies are observed to contract SCID disease as a newborn. This occurs due to a mutation in the mutation of the ADA gene which is responsible for stopping the activity of the enzyme adenosine deaminase. This adenosine deaminase enzyme is necessary to maintain a healthy immune system.
This disease is usually fatal within 2 to 3 years of birth if left untreated. This disease is usually associated with the immune system making the person vulnerable to many diseases which end up being fatal.
The director of the NIH’s National Institute of Allergy and Infectious Diseases (NIAID) said that, according to research, gene therapy could potentially help overcome this disease in newborns as well as children. In addition, this gene therapy is a one-time treatment after which a person can live a healthy life with an excellent immune system.
There are other ways to treat this disease as well, but no treatment works as gene therapy. Other treatments include, enzyme replacement therapy, hematopoietic stem cell transplants, etc.
The latest experiments suggest that lentiviral gene therapy is the safest of all existing gene therapies and gives better results than others. This procedure involves the insertion of the ADA gene into the patient’s own hematopoietic stem cells.
These have been shown to be effective and constitute a one-time therapy that would certainly save the lives of millions of people.