Scientists at the Ecole Polytechnique Fédérale de Lausanne (EPFL) in Switzerland have used RNA interference in transgenic mice to silence a mutated gene that causes inherited cases of amytrophic lateral sclerosis (ALS), substantially delaying both the onset and the progression rate of the fatal motor neuron disease. Their results will be published in the April issue of Nature Medicine, and in the journal’s advanced online publication March 13. In addition to silencing the mutated gene that causes ALS, the EPFL researchers were able to simultaneously deliver a normal version of the gene to motor neuron cells using a single delivery mechanism. “This is the first proof of principle in the human form of a disease of the nervous system in which you can silence the gene and at the same time produce another normal form of the protein,” notes Patrick Aebischer, EPFL President and a co-author of the study.
