Animal models of amyotrophic lateral sclerosis.

Animal models of human disease are important in unravelling the pathophysiology 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 the paucity of knowledge of their aetiology and the organizational specificity of the human motor system. Nonetheless, a range of spontaneously occurring, 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 particularly true for the transgenic mouse models expressing superoxide dismutase or cytoskeletal proteins. This approach has provided an unparalleled opportunity 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.