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October 23, 1991

Gene Replacement Therapy Enters Second Year

JAMA. 1991;266(16):2193. doi:10.1001/jama.1991.03470160025008

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AT THE START of the second year after initial clinical use of gene replacement therapy, the patient is "doing exceptionally well," says R. Michael Blaese, MD, National Cancer Institute who, with W. French Anderson, MD, National Heart, Lung, and Blood Institute, is one of the principal National Institutes of Health (NIH) clinicians involved in the study (JAMA. 1991; 265:2311-2312).

The patient, a 5-year-old girl, has adenosine deaminase (ADA) deficiency, a rare genetic disorder that paralyzes the immune system. On September 14,1990, at the NIH in Bethesda, Md, she received the first of eight infusions of her lymphocytes, cocultivated in the presence of a retroviral vector carrying the missing ADA gene. Some percentage of the lymphocytes pick up the gene and the gene-corrected lymphocytes are reinfused back into the patient.

For 2 years before the patient became the first to receive gene replacement treatment, she had been receiving enzyme replacement with