genetically modified rodent, cell or tissue, methods for producing a genetically modified rodent and motor neuron population, methods for selecting a candidate agent to modulate motor neuronal dysfunction and a candidate agent for reducing oxidative stress in a motor neuron, nucleic acid and rodent cell?

摘要

The present invention relates to an animal model for motor neuronal dysfunction in diseases, for example amyotrophic lateral sclerosis (ALS), comprising a genetically modified non-human animal comprising a genetically modified dr6 allele and displaying normal phenotypes at birth. and for a few weeks or months after birth. however, as the nonhuman animal ages, it develops motor neuronal dysfunction that presents as one or more symptoms of als, which may progress rapidly after onset. Methods for identifying candidate agents that can be used to prevent, delay or treat als are also provided.