width=With funding from the A-T Children’s Project, the laboratory of Anthony Wynshaw-Boris, MD, PhD at Case Western Reserve University in Cleveland, Ohio will attempt to grow A-T brain cells in laboratory dishes to overcome the obstacle of not being able to access and study living brain cells from A-T patients.

Dr. Wynshaw-Boris knows A-T well. In 1996, he led the first scientists to genetically engineer an A-T mouse model, and he knows the limitations of using A-T mice to understand the disease in humans. Mouse models have lacked the progressive neurological problems that patients with A-T have, preventing scientists from learning how brain cells become sick and die, causing the movement problems of A-T.

Using cutting-edge stem cell and DNA editing technologies, including CRISPR/Cas9, Dr. Wynshaw-Boris and his team will change skin cells obtained from patients with A-T into “induced pluripotent stem cells”, and then into brain cells, to explore how the loss of the A-T protein causes so much harm. It’s our hope that these new cell models will lead to the identification of drug targets and effective therapies for A-T.

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