Abstract
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.
Acknowledgements
The authors would like to thank the researchers who deposited their models into The Jackson Laboratory, and especially A Burghes for all of the outstanding models he generously made available to the spinal muscular atrophy community. Finally, a special thanks is given to D Kobayashi for her helpful advice and support over the years.
Financial & competing interests disclosure
The authors would like to acknowledge funding from the SMA Foundation, the Friedreich‘s Ataxia Research Alliance, the ALS Association, the Tow foundation and the ALS Therapy Foundation, and for their support in funding disease-specific mouse repositories. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
No writing assistance was utilized in the production of this manuscript.