New Transgenic Technologies

Transgenic tools for genome modification can be used to produce animal models of human movement disorders. Genetic changes that cause disease can be introduced into mouse and rat chromosomes with great facility by a number of methods, including transgenesis that adds genes, gene-targeting in embryonic stem cells that produces conditional alleles, and nuclease-mediated gene knockouts and knockins. The use of inducible transgene expression in combination with Cre/loxP technology provides control over gene expression so that the timing of gene expression or ablation during the life of an animal is under experimental control. Next-generation sequencing methodologies and genome-wide association studies provide processes for the continuous discovery of candidate genes responsible for human disease. Mutations found in patients can be replicated with great facility in mouse and rat models. The challenge for the future is translating findings in animal models into treatments that ameliorate illness.