Gene therapy saves immune deficient babies in France
Gene therapy has been successfully used by French researchers to treat severe combined immunodeficiency (SCID). Two babies, aged 8 and 11 months, who received the treatment are no longer in isolation and live at home without any treatment, enjoying normal growth and development for their ages.
The normal functioning of the infants' immune system has lasted 11 months without any side effects so far, and a third patient is making similar progress four months after the gene transfer.
Usually patients with SCID are required to live within tightly controlled and sterile areas to avoid any threats to their non-functioning immune system until a bone marrow transplant is attempted. The new treatment, described in Science (2000; 288:627-9, 669-72), involved providing a normal copy of the defective gene that causes SCID X1, a life threatening disease inherited on the X chromosome.
The defective gene encodes part of a cell receptor that sends