We take the patient’s own blood cells (ethically derived and minimally invasive) and turn them into a different cell type called an induced pluripotent stem cell (iPSC). We then turn the iPSCs into muscle cells since these are a disease-relevant cell type. Patient-derived muscle cells can then be grown in a single layer (called 2D culture) and used to advance our research, by modelling the features of the disease in a dish. We are actively exploring relevant disease biology using bulk transcriptomic, proteomic, single cell and single nuclei approaches on patient-derived cells, as well as control cells and a range of tissues. Cultured cells are an extremely useful resource but do have limitations in 2D since they are not identical to the more complex skeletal muscle in our bodies that allows us to move, eat and breath. To keep improving the capabilities of these cells to model patient disease features (such as muscle strength) we are working with a leader in the developmental biology field, Professor David Mack at the University of Washington, Seattle. We are working with David Mack’s team to grow contractile patient muscle cells in 3D, enabling their maturation and the measurement of muscle contractile force. This is a really exciting avenue of our research that we are actively pursuing at the moment. We are also exploring advanced ‘humanised’ in vivo models. Our hope is that by establishing robust methods for screening treatments in 2D and 3D cell cultures, as well as in vivo, we will have more power to identify promising treatments with real potential to improve patient muscle function. Therefore, patient-derived muscle cells are also essential for the second aim of the Team – developing treatments.