Cedars-Sinai researchers have identified a potential new therapeutic strategy for combating Lou Gehrig's disease, a fatal disorder that attacks muscle-controlling nerve cells in the brain and spinal cord.
According to a report on the Medical Express
Website, the researchers used an innovative stem cell technique to create brain cells in a lab dish from skin scrapings of patients with the disorder, then inserted genetic material that blocked the damaging effects of a defective gene involved in the condition.
The research, published in the journal Science Translational Medicine, is among the first to create a specific form of Lou Gehrig's disease (amyotrophic lateral sclerosis, or ALS) in a dish and then treat it — marking a significant step toward a future therapy.
"In a sense, this represents the full spectrum of what we are trying to accomplish with patient-based stem cell modeling," said lead researcher Robert H. Baloh, M.D., with the Cedars-Sinai Regenerative Medicine Institute. He is the lead researcher and the article's senior author.
"It gives researchers the opportunity to conduct extensive studies of a disease's genetic and molecular makeup and develop potential treatments in the laboratory before translating them into patient trials."
The researchers found that genetic defects may cause the disease and that gene therapies may be the most effective way to treat it.