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Article: Mouse Model May Help Better Quantify Gene Therapy Risks.
- Article from:
- BIOWORLD Today
- Article date:
- November 3, 2009
CopyrightCOPYRIGHT 2009 A Thomson Healthcare Company. This material is published under license from the publisher through the Gale Group, Farmington Hills, Michigan. All inquiries regarding rights should be directed to the Gale Group. (Hide copyright information)
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Science Editor
Knowing that a risk exists is one thing. But knowing the odds is something very different.
Take gene therapy. In 2000, 11 baby "bubble boys" with x-linked severe combined immunodeficiency disorder (X-SCID) were treated by gene therapy in a clinical trial at the pediatric Hospital Necker Enfants Malade in Paris. The boys were treated with blood stem cells from their own bone marrow, which were modified ex vivo to contain the protein they were missing: a functioning interleukin-2 receptor gamma chain. At the time, the intervention was hailed as gene therapy's first stunning success. (see BioWorld Today, May 1, 2000.)
But three of ...