IN VIVO SCREENING MODELS FOR TREATMENT OF isoQC-RELATED DISORDERS

摘要

The present invention provides a transgenic non-human animal, in particular a transgenic mouse encoding isoQC, which has been implicated in isoQC-related diseases. The present invention additionally provides cells and cell lines comprising transgenes encoding for isoQC. The present invention further provides methods and compositions for evaluating agents that affect isoQC, for use in compositions for the treatment of isoQC-related diseases.