Genetics news & views from students enrolled in BIOL 2110 at Stockton University.
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.