The A-T Children’s Project has granted funding for Yang Xu, PhD from the University of California, San Diego to continue development of a stem cell-based model for A-T. This type of “disease-in-a-dish” model can be used to screen hundreds of thousands of compounds for their ability to diminish or reverse certain characteristics of the disease in culture. Promising compounds or drugs can then be developed for future clinical trials.

Stem cells in particular are proving to be useful tools for disease research, because they can be stimulated to turn into various other cell types found in the body. They are especially useful models for neurological disorders, as brain cells cannot be obtained for research purposes from living patients.

In December 2007, the A-TCP provided funding to Dr. Xu to develop stem cells in which the A-T protein (ATM) could be turned off at various stages during their transition (or differentiation) into other cell types, like brain cells. These types of cells are called ‘conditional’ stem cells, because the ATM protein can be turned off only under certain experimental conditions.

Though technically challenging, Dr. Xu successfully generated these conditional ATM stem cells. The A-TCP is now funding Dr. Xu’s laboratory to characterize the new cells and use them to investigate the mechanisms of brain cell death in A-T and to identify chemical compounds that could potentially prevent brain cell loss in patients with this disease.

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