Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating neurodegenerative disease. “Silencing” a gene associated with regulating TDP-43, the protein that accumulates in the brain and causes ALS, with a technique called RNA interference (RNAi) allowed mice to survive an average of 54 percent longer. Subjects also experienced improvements in strength and reduced inflammation in the brain and spinal cord, according to research from the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia, published today in Nature Communications.
