Two More Cell Infusions on Schedule for Gene Replacement Therapy Patient

Abstract

EIGHT MONTHS after beginning treatment, the first patient to be given gene replacement therapy is making what researchers consider to be significant progress. The patient is a 4-year-old girl with adenosine deaminase (ADA) deficiency. She is being treated at the National Institutes of Health (NIH), Bethesda, Md. The NIH research group plans to perform two more cell infusions on this patient. At that point, says Michael Blaese, MD: "We will make a detailed study of other indicators of T-cell reconstitution and immune function. We haven't progressed as rapidly as we would have liked, but nevertheless, I am delighted at the way things are going." <h3>Gene Being Expressed</h3> At this point, there is evidence that the ADA gene that corrects for the deficiency is being successfully inserted into the patient's lymphocytes, albeit at a very low rate (between 3% and 8%). Laboratory findings indicate that the gene is being expressed; ADA