Science / Medicine : 2 Research Groups Develop Gene Therapy

A new approach to gene replacement therapy in animals and, eventually, in humans has been developed independently by two groups of researchers. The groups reported last week in the journal Science that they had used a special virus to insert the gene for a protein into cells isolated from the lining of blood vessels, then reintroduced the cells into pigs and dogs. The engineered cells then colonized the interior of blood vessels and began producing the new protein.

In the past, researchers have speculated about using bone marrow cells, the source of blood cells, for gene replacement therapy. The new technique should be simpler to perform and less painful for the the recipient.

The researchers said that the first use of the technique might be to introduce anticoagulants, vessel-dilating or growth-promoting agents into the vascular system. The procedure also could be used for the treatment of any inherited disease that requires secretion of biological agents directly into the bloodstream.