Guidelines for Gene Therapy

The approval of federal guidelines for gene-transplant experiments on humans is an important step in realizing the potential of genetic research.

Under the guidelines, researchers who receive federal funds must submit a detailed proposal for each genetic therapy that they want to undertake, and must receive approval from a local committee and from the Recombinant DNA Advisory Committee of the National Institutes of Health in Washington.

At the moment, all research in this area is federally supported. If commercial firms were to enter the field, they would almost certainly comply with the guidelines, just as they voluntarily comply with other federal guidelines on genetic engineering.

Now that guidelines exist, proposals are expected to be forthcoming within the next few months to conduct gene-therapy experiments on children with a rare and fatal enzyme deficiency caused by a faulty gene. This disease, called adenosine deaminase deficiency, frequently leads to death in children by age 2.

Doctors will attempt to remove bone marrow from the child, transplant healthy genes into the bone-marrow cells and put them back into the patient’s body. The transplanted genetic material will not be inherited by the next generation. As a result, gene therapy of this kind “does not differ fundamentally from other kinds of conventional medical care--it’s another kind of transplant,” according to Dr. LeRoy Walters of the Center for Bioethics at Georgetown University, who chaired the committee that drew up the guidelines.


From the beginning, genetic research has raised understandable concern about a variety of medical, social and ethical problems. But medicine has learned enough about genes and how they work that it is ready to treat human patients for whom no other treatments are available.

The federal guidelines are thorough and thoughtful. They will enable people in need of treatment to receive it.