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Models of human disease are essential in the discovery of new therapies. Cell based models are often designed to provide a platform for high-throughput drug screening, typically involving rapid fluorescence or other optical readout related to some aspect of the pathology, which can then serve as a basis for a high-throughput screen. However, drugs identified in cell models rarely progress to the clinic. Animal models, although sometimes poorly suited to high-throughput screening, have two major advantages. Firstly, cell and tissue interactions are in place. Secondly, they enable details of drug delivery and pharmacokinetics to be explored, thereby approaching more closely the therapeutic goal.Ìý
The aim of this module is to provide you with a sound understanding of the genetic model organisms available for studies relevant to human disease. These model organisms include the yeast Saccharomyces cerevisiae, the nematode Caenorhabditis elegans, Drosophila melanogaster (fruit fly), Danio rerio (zebrafish), and Mus musculus (house mouse).Ìý
Upon completion of this module, you will have obtained insights and understanding of;

•ÌýÌý Ìýthe biology of the genetic model organisms studied and their potential for studying human disease.
•ÌýÌý Ìýexperimental approaches utilised in the investigation of the mechanisms of disease and exploring therapies for those diseases.Ìý
•ÌýÌý Ìýthe generation and deployment of mutants and transgenic lines important in the study of human disease.Ìý
•ÌýÌý Ìýcontribution of various model organisms in understanding the underlying mechanisms of disease, and understanding the challenges of mimicking human diseases in model organisms.
•ÌýÌý Ìýthe utility of genomics and the applications of forward and reverse genetic screens.Ìý
•ÌýÌý Ìýthe drug discovery process, with examples of successes from small molecules through to antibodies and other biologics.

As well as lectures, students will gain first-hand experience with the model nematode, Caenorhabditis elegans, and observe how they can be deployed in phenotypic drug screening, genetic screening to search for new drug targets, and in chemistry-to-gene screens to identify the pathway targeted by a drug.
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