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Gene therapy may offer release from sterile isolation for patients lacking immune systems Part 1 (Teacher: Michael)

A new gene therapy method has turned the bone marrow of two young children from an "immune cell desert" into a healthy breeding for a variety of infection-fighting agents.

The children have a form of severe combined immuno-deficiency disorder (SCID) that typically requires patients to stay in sterile environments. They are now living and developing normally, Italian and Israeli researchers report in the journal Science, published by the American Association for the Advancement of Science.

Specifically, the two children have a form of the disease called ADA-SCID, in which they not produce the ADA enzyme necessary for making immune cells. The disease can controlled to a certain degree by regular injections of the bovine form of ADA, an expensive proposition in many countries.

From 30 to 50 infants with ADA-SCID are each year, but only a small portion of them are diagnosed, according to study co-author Claudio Bordignon of the San Raffaele Telethon Institute for Gene Therapy and Università Vita-Salute San Raffaele in Milan.

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