In the USA, UCLA Mattel Children’s Hospital physicians have been reported to have treated 48 of 50 children without immune system who were born with a rare and fatal hereditary disease with gene therapy, which they developed by taking advantage of AIDS.
The leader of the clinical trial, Dr. Donald Kohn said in a statement to the press that “they took a potentially fatal disease and cured most of the children with a single treatment.”
CHILDREN WITHOUT IMMUNITY SYSTEM HAVE TO LIVE INSULATED
It was stated that the treated children had severe combined immunodeficiency syndrome or SCID caused by an inherited genetic defect, and this situation prevents the bone marrow that makes up the immune system from making healthy versions of blood cells.
It was stated that if such patients are not treated, they usually die in the first or two years of their lives, while it was stated that children born with this disease should live in a plastic balloon or room in order to be isolated from germs.
In the gene therapy developed by doctors, some of the stem cells that form blood are taken from the patient without an immune system, and then a new copy of the ADA gene is sent to the stem cell by an inactivated AIDS virus.
The corrected cells are then sent back to the child’s body to continuously produce healthy immune cells that can fight infection.
Results of the UCLA-led study were published by the New England Journal of Medicine and presented at the American Society of Gene & Cell Therapy conference online.