Washington: Researchers have developed a therapy that could dramatically slow the onset and progression of the Lou Gehrig’s disease.The researchers, led by teams from The Research Institute at Nationwide Children’s Hospital and the Ludwig Institute at the University of California, San Diego, found a survival increase of up to 39 percent in animal models with a one-time treatment, a crucial step toward moving the therapy into human clinical trials.The therapy reduces expression of a gene called SOD1, which in some cases of familial amyotrophic lateral sclerosis (ALS) has a mutation that weakens and kills nerve cells called motor neurons that control muscle movement.While many drug studies involve only one type of animal model, this effort included analysis in two different models treated before and after disease onset.Brian Kaspar, PhD, a principal investigator in the Center for Gene Therapy at Nationwide Children’s and a senior author on the research, said that they designed these rigorous studies using two different models of the disease with the experimenters blinded to the treatment and in two separate laboratories.
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