Optimizing mouse models of neurodegenerative disorders: are therapeutics in sight?

Future Neurol. 2013;9(1):67-75. doi: 10.2217/fnl.13.66. Epub 2013 Dec 16.

Abstract

The genomic and biologic conservation between mice and humans, along with our increasing ability to manipulate the mouse genome, places the mouse as a premier model for deciphering disease mechanisms and testing potential new therapies. Despite these advantages, mouse models of neurodegenerative disease are sometimes difficult to generate and can present challenges that must be carefully addressed when used for preclinical studies. For those models that do exist, the standardization and optimization of the models is a critical step in ensuring success in both basic research and preclinical use. This review looks back on the history of model development for neurodegenerative diseases and highlights the key strategies that have been learned in order to improve the design, development and use of mouse models in the study of neurodegenerative disease.

Keywords: ALS; Friedreich’s ataxia; SMA; genetic background; mouse models; neurodegeneration; preclinical trials.