ALS subject of "disease in a dish".

• Position: Lou Gehrig's Disease - Brief article

Although the technology has existed for just a few years, scientists increasingly use "disease in a dish" models to study genetic, molecular, and cellular defects, but a team of doctors and scientists led by researchers at the Cedars-Sinai Re generative Medicine Institute, Los Angeles, Calif., went further in a study of Lou Gehrig's disease, a fatal disorder that attacks muscle-controlling nerve cells in the brain and spinal cord.

After using a stem cell technique to create neurons in a lab dish from skin scrapings of patients who have the disorder, the researchers inserted molecules made of small stretches of genetic material, blocking the damaging effects of a defective gene and, in the process, providing "proof of concept" for a new therapeutic strategy--an important step in moving research findings into clinical trials.

The study, published in Science Translational Medicine, is believed to be one of the first in which a specific form of amyotrophic lateral sclerosis was replicated in a dish, analyzed, and "treated," suggesting a potential future...