The first successful treatment of a genetic disorder by gene therapy was the curing of a 4-year-old girl who had severe combined immunodeficiency disease. Because the engineered cells did not live forever, this child supplemented her treatment with regular doses of

Question

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The girl had severe combined immunodeficiency disease due to a deficiency in the enzyme adenosine deaminase (ADA). Regular doses of ADA were used to supplement her treatment.

The First Successful Treatment of a Genetic Disorder by Gene