UCL HUNTINGTON’S DISEASE RESEARCH
KERATINOCYTE-DERIVED INDUCIBLE PLURIPOTENT STEM CELLS IN HUNTINGTON’S DISEASE (K-IPS-HD)
Cells can be grown in a dish, providing a powerful means of studying the fundamental basis of a disease and for screening potential drugs. The current animal based cell models of HD have limitations and are far from optimal as they do not entirely recreate the human disease.
Cell lines generated from patient material that can be differentiated into neurons promise to provide much more relevant models to better understand how the disease works as well as a means for testing drugs in the future.
Using hair samples from control and HD subjects and in collaboration with Professor Gillian Bates’ team at King’s, we will extract hair cells which will be turned into stem cells and from there into neurons. We will then be able to compare the behaviour of HD neurons to normal neurons to better understand the steps that are important for initiating HD. We can also use the same technique to study the behaviour of other cells that are affected in HD, e.g. muscle cells.
Due to methodological difficulties, this study has finished early.