This research utilizes the ability to reprogram cells to devise new treatments for clinical injuries and diseases that currently have no useful treatment options. Work includes both in vivo and in vitro projects using combinations of transcription factors and small molecules to change the phenotype of one cell into another. Generating the clinically useful cell type can be a direct, in vivo reprogramming event as in a gene therapy for diabetes where a three-gene combination stably converts liver cells into functional insulin-secreting cells to restore blood glucose homeostasis in diabetic animal models. Althernatively, the researchers may utilize a pluripotent stem cell intermediate, such as induced pluripotet stem cells (iPSCs), that are subsequently differentiated to generate the therapeutic cell product. This approach is how they are investigating the clinical manufacture of cells such as oligodendrocyte progenitor cells (OPCs) to treat spinal cord injury and other dysmyelinating diseases, retinal pigmented epithelial (RPE) cells to prevent vison loss due to age-related macular degeneration, and other iPSC-derived cells such as NK cells to treat cancer and cells to treat debilitating skin diseases and wounds.
MSI use encompasses two aspects of this work. Transcriptome analysis using RNA-seq or hybridisation array data is performed to understand cell type identity and analyze the progression and outcomes of reprogrammng and differentiation experiments. In addition the researchers are planning to incorporate exome sequence comparisons in the clinical iPSC manufacture SOPs and are currently working to generate a feasible work flow for this.