Animal models of amyotrophic lateral sclerosis

Animal models of human disease are important in unravelling the pathophysio logy of the condition, for exploring the natural history of disease and for evaluating potential therapies. The development of animal models of human neurodegenerative disease such as ALS is particularly challenging, given th e paucity of knowledge of their aetiology and the organizational specificit y of the human motor system. Nonetheless, a range of spontaneously occurrin g, experimentally produced, or genetically engineered models of ALS are now available. Although not always a perfect replica of the ALS disease, these models are shown to be of outstanding importance for investigations of the mechanisms of dysfunction/death of motor neurons in vivo. This is particul arly true for the transgenic mouse models expressing superoxide dismutase o r cytoskeletal proteins. This approach has provided an unparalleled opportu nity for testing of potential pharmacological or gene therapies, and it can be expected that the results of these studies will be translated into the clinical advances of the next years.