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Gene therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID)

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Gene therapy has had a long gestation; the concept is almost 40 years old. Initially, attention centred on the haemoglobinopathies, but technical difficulties led to disappointment. Subsequently, interest turned to other diseases including the severe combined immunodeficiencies (SCIDs). The results of attempts to cure adenosine deaminase (ADA)-deficient SCID in the early 1990s were again disappointing, but technical improvements were followed by reports of more favourable results in both ADA-deficient and X-linked SCID in the early years of this century. Now the long-term results of gene therapy in 10 children with ADA-deficient SCID have been reported …

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