Most neurodegenerative diseases remain untreatable and for the common diseases we still have little idea of the underlying biological mechanisms. In the new mouse models of neurodegeneration laboratory (MMON) at Harwell we are using novel and innovative genome engineering techniques to create bespoke models to help answer key questions in neurodegenerative disease.
In collaboration with colleagues at the UCL Institute of Neurology who work on amyotrophic lateral sclerosis, frontotemporal dementia and Down syndrome and Alzheimer’s disease, we are designing novel animal models, with an emphasis on gene expression at physiological levels. These new models will undergo phenotyping at Harwell, using the expertise across many modalities that characterises the excellent Harwell phenotyping program. In parallel we and our expert collaborators at UCL will carry out in depth phenotyping specifically analysing behaviour, neuronal and muscle function, to answer important questions about the mechanisms of neurodegeneration, with a view to building up platforms for testing therapies, including small molecule and gene therapies.
This is an exciting collaboration in genome engineering and neuroscience, between our group, run by Edgar Kramer in collaboration with Elizabeth Fisher (Harwell and UCL), and neuroscientists and clinicians at UCL including Pietro Fratta, Linda Greensmith, Adrian Isaacs, Giampietro Schiavo, and Frances Wiseman.
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