BETHESDA, MD. — National guidelines were approved Monday for a revolutionary type of medical treatment to be used on humans against a range of fatal hereditary diseases.
The treatment, known as gene therapy, involves transplanting genes -- the body's basic blueprints for cell construction -- to correct genetic defects that cause these illnesses.
Monday's approval by a government advisory panel came after more than seven months of discussion and public comment.
Under the guidelines, every case in which gene therapy is proposed must still be approved by local review committees and the federal government.
Each planned therapy, the first of which is expected to be proposed within a matter of months, must conform strictly to the specific guidelines that were given final approval on Monday.
The first diseases on which gene therapy is likely to be used are rare hereditary conditions that leave the patient vulnerable to infection and disease.
The guidelines were approved unanimously by the Recombinant DNA Advisory Committee of the National Institutes of Health at the committee's fall meeting. Scientists at the committee meeting said the vote marks an important step toward bringing this kind of treatment into practical use.
Now that guidelines have been established, the several scientific teams studying gene therapy can proceed to proposing specific trials with patients. The federal rules forbid any attempt at gene therapy by any institution receiving federal research money without approval by the National Institutes of Health. All of the research teams known to be considering such experimental treatments are affiliated with institutions that do receive such federal support.