PUBLIC RELEASE DATE: 14-Aug-2014 Contact: Mary Beth O'Leary moleary@cell.com 617-397-2802 Cell Press By targeting RNA molecules that tangle and clump in the nervous systems of patients with the most common genetic form of amyotrophic lateral sclerosis (ALS or Lou Gehrigs disease) and frontotemporal dementia (FTD), researchers have shown they can effectively limit those damaging elements in cells taken from patients. The results reported in the Cell Press journal Neuron on August 14th show that RNA is a viable drug target for the two overlapping and incurable neurodegenerative diseases. The abnormal proteins derived from that aberrant RNA might also serve as biomarkers in clinical trials to test the new ALS and FTD drug candidate and otherwise monitor the diseases, the new study finds. ALS is caused by a progressive loss of motor neurons, leading to severe impairment of mobility, speech, swallowing, and respiratory function that is usually fatal within two to five years, the researchers explained. In FTD, brain regions that support higher cognitive function are affected instead to produce disabling changes in behavior, personality, and language. At present, no effective treatment is available for either condition. However, experts believe that a better understanding of the underlying disease processes will expedite … Continue reading
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