WASHINGTON — The first patient to be treated with gene therapy, a child suffering from an immune deficiency, is responding and growing more healthy, a researcher said Monday.
The patient, identified only as a 4-year-old girl, was injected last September with genetically altered cells designed to correct a deficiency of an enzyme essential to the immune system.
Dr. R. Michael Blaese, a National Institutes of Health scientist and a co-researcher in the experiment, said at an American Assn. for the Advancement of Science meeting that the child has received four infusions of cells altered to contain a missing gene.
The gene causes the secretion of an enzyme called adenosine deaminase, or ADA. The child was born without this gene. Such children do not develop an immune system and usually die of infection by age 2.
The doctor said the child's ADA levels are now about 20% of normal and there is a "striking increase" in isohemagglutinin, a component of the blood that is a measure of the immune system.