Article: Mouse Model May Help Better Quantify Gene Therapy Risks.

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 ...

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