Gene therapy may offer release from sterile isolation for patients lacking immune systems

This news release is also available in French and Italian

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

The children have a form of severe combined immunodeficiency 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 do not produce the ADA enzyme necessary for making immune cells. The disease can be controlled to a certain degree by regular injections of the bovine form of ADA, an expensive proposition in many countries.

Approximately 30-50 infants with ADA-SCID are born 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 Universit Vita-Salute San Raffaele, in Milan.

Bone marrow transplants have also been successful in some cases, but they pose serious risks of their own. There was no available donor for the two children in the study, who were seven months old, and two years and six months old.

"With gene therapy you can treat every patient, and the toxicity is enormously lower than for bone marrow transplants. We propose, at this stage, that every patient lacking a donor with an identical tissue type should be directed to gene therapy," said Bordignon.

Bordignon's team must test their therapy on more volunteers before it will be approved for researchers to use in Italy or abroad. They will start with p

Contact: Lisa Onaga
American Association for the Advancement of Science

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