GENOME EDITING OF GENES ASSOCIATED WITH TRINUCLEOTIDE REPEAT EXPANSION DISORDERS IN ANIMALS

摘要

The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with trinucleotide repeat expansion disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.