Friday, July 30, 2021

Rescuing Children Born Without Immune Systems With HIV Gene Therapy Methods

    Experimental gene therapy leads to 48 out of 50 young children born without immune systems being able to acquire an essentially normal immune system.  Children born without an immune system can experience life-threatening illnesses from simple day-to-day activities. 50 of the young children had a form of immune deficiency called ADA-SCID. This is where a faulty gene causes a buildup in the bloodstream with a common and natural biochemical called adenosine. By utilizing experimental gene therapy inside a newborn's body the genetic defect that causes immune deficiency is able to be fixed. The procedure uses a modified, harmless version of AIDS-causing human immunodeficiency virus (HIV) in order to cure the patient.  During the procedure, doctors extract some of a child's bone marrow and expose it to a modified version of HIV containing a cloned, normal copy of their broken gene. "We collect blood-forming stem cells from the bone marrow of these patients, bring them to the laboratory, use this lentiviral vector to insert this normal gene into their stem cells, and then they're given back to the patients by intravenous infusion," said lead researcher Dr. Donald Kohn. Then children undergo chemotherapy to destroy their faulty bone marrow, and the new genetically modified cells are put back in as a replacement. Children who have had this procedure have not needed any other medical assistance pertaining to their previous immune deficiency after the gene therapy.

Helpful Links:

Gene Therapy Uses HIV to Rescue Kids Born Without Immune System

Adenosine deaminase deficiency

By Katherine Morone


  1. This experimental gene therapy seems to have yielded remarkable results. Perhaps this gene therapy will be used more widely as the children who have undergone this procedure have not needed any medical assistance regarding their previous immune system deficiencies.

  2. I think the field of experimental gene therapy is very interesting and I found your post very informative. It is fascinating how scientists are able to extract and alter stem cells from bone marrow and then re-insert them into the host to help cure genetic defects. There are a lot of possible uses for this procedure that can help with many genetic disorders.

  3. Gene therapy seems to be a great tool for doctors and geneticists around the world. I remember learning in previous courses that it has great impacts on a multitude of diseases. I think it is amazing that these children are able to be helped with their illness, because back when this technology wasn't discovered, these children would ultimately have a fatal illness.

  4. Gene therapy is an extremely interesting topic and this post was very informative. I learned that doctors can extract cells and use them to treat genetic illnesses. It is interesting to see the advance in technology that allows for treatment of these conditions.