Top 10 Promising Gene Therapies : Discovery Tech : Discovery Channel

The Wide Angle: Top 10 Promising Gene Therapies

By Vicki Miskovsky

Bubble Boy Disease

David Vetter, shown here on September 11, 1982, who lived with SCID from within the protective confines of a plastic bubble, was the subject of movies about his life. He died in 1984, from complications of an experimental bone marrow transplant.

1. Severe Combined Immunodeficiency Disorder (SCID)

In 1990 SCID, a rare but life-threatening disease, became the first human disorder ever to be treated with gene therapy. Known as "bubble boy" disease because of the plastic barriers used to protect vulnerable patients from infections, SCID results from a missing gene that makes a protein essential for effective function of the immune system.

There have been gene therapy trials for two types of SCID, X-linked and ADA (adenosine deaminase) deficiency SCID. Results reported in the January 29, 2009 issue of The New England Journal of Medicine show eight out of ten ADA deficiency SCID patients and 18 of 20 X-linked SCID patients had immune recovery following gene therapy. Carter calls this a durable cure that allows kids to go out in the world and not worry. This therapy is now available in Europe.

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The Wide Angle: Top 10 Promising Gene Therapies

Bubble Boy Disease

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