Disease in a Dish
Generating cells from patients suffering from such disorders as Down syndrome, schizophrenia, and ALS lets scientist study these diseases in the lab and test new drugs.
Ricardo Dolmetsch and collaborators from Stanford created neurons from a patient with Timothy syndrome, a rare inherited disorder that affects both the heart and the brain and can cause autism. Growing patient-derived neurons (above, red) in a dish, the scientists discovered that the cells were making too much of an enzyme (shown in green) that is critical for producing dopamine and norepinephrine, two important chemical messengers in the brain. Treating the cells with a specific drug blocked some of the excess.
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