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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 bone marrow of two young children from "immune cell desert" into healthy breeding ground for variety of infection-fighting agents.

The children have 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 journal Science, published by American Association for Advancement of Science.

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

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

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