The NeuroGenetics Center has the goal of creating non-human primate models of neurodevelopmental, neuropsychiatric and neurodegenerative disorders. The use of non-human primate models is likely to greatly accelerate the development of new cures and treatments for neural disorders, because the genetics and brains of non-human primates are so similar to those of humans.
Historically, rodents have been the most widely used animal model in neuroscience laboratories. However, these animals have proven to be of limited value in uncovering new treatments or prevention of disorders in people. Perhaps this should not be surprising; while the use of transgenic mouse lines has yielded considerable insights into normal and abnormal brain function, the rodent nervous system differs in fundamental respects from the human nervous system. The growing recognition of these crucial differences, particularly those that enable movement and higher level cognitive and social behaviors, has led to rising interest in the development of transgenic models in non-human primates. Marmosets are increasingly favored in this respect, because they are easily handled in a lab setting, and reproduce at a rate high enough to make it feasible to create transgenic models of disorders.
In the fall of 2014, the Brain Institute opened a research facility devoted to the study of neural development in freely behaving marmosets. Faculty members who are expert in the use of genome-editing techniques, including CRISPR/Cas9 and TALEN, aim to use these techniques to establish marmoset models of a variety of specific neurological disorders. Creation of these models will be a first step toward ameliorating and even preventing these conditions in people. Initial targets include amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and certain ataxias.