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Gene therapy frees two children from sterile 'bubbles,' Science authors report

Note: This news release is available in French and Japanese .

Washington D.C.- French researchers have developed a method of gene therapy to treat human severe combined immunodeficiency (SCID) X1, a life-threatening disease inherited on the X chromosome. Usually, patients with SCID are forced to live within tightly controlled and sterile "bubbles" to avoid any threats to their nonexistent immune systems until a bone marrow transplant is attempted. The new therapy is described in the 28 April issue of Science.

Two infants, aged 8 and 11 months, were the beneficiaries of this treatment, which provides a normal copy of the defective gene that causes SCID X1 that quickly proliferates within the patient's body. The new gene "unblocks" the development of other immune cells, restoring the immune system to normal functioning. The infants' return to a normal immune system has lasted over eleven months without side effects, says study co-author Alain Fischer of the Hospital Necker in Paris. The Science report notes that a third patient is experiencing similar progress four months after the gene transfer.

The defective gene encodes part of a cell receptor that sends out signals to the parents of T and NK cells, crucial components of the immune system that destroy invaders and rally other immune defenses. Without this gene's direction, these cells do not develop, grow, or spread, and SCID X1 patients are left fatally vulnerable to even slight infectious insults to the body such as a cold sore or common childhood diseases like chicken pox.

The researchers began the therapy by harvesting bone marrow from the patients and sorting out a set of blood stem cells from the marrow. After bathing in a growth factor in containers coated with a fibronectin fragment, a threadlike pr
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Contact: Heather Singmaster
hsingmas@aaas.org
202-326-6414
American Association for the Advancement of Science
27-Apr-2000


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