TRANSGENIC ANIMAL MODEL FOR NEURODEGERATIVE DISEASE AND USES THEREOF

摘要

The invention provides a transgenic non-human mammal comprising nucleated cells containing a transgene encoding an ataxin-2 polypeptide comprising a polyglutamine tract. In particular, the transgenic-non-human mammal can be a mouse. The invention also provides methods of using a transgenic non-human mammal expressing SCA2 encoding an ataxin-2 polypeptide to identify a therapeutic agent for use in treating a neurodegenerative disease by administering a compound to a transgenic non-human mammal expressing SCA2 encoding an ataxin-2 polypeptide and screening the transgenic non-human mammal for an improved neurological response associated with a neurodegenerative phenotype of the transgenic non-human mammal, thereby identifying a therapeutic agent for use in treating the neurodegenerative disease.