Regenerative medicine for diabetes: automated gene circuits for therapeutic beta cell production

Type 1 diabetes is a serious autoimmune disease in which the body's immune system destroys the insulin-producing β-cells in the pancreas. Millions of people are therefore dependent on a lifelong supply of insulin, and there is currently no cure. Transplantation of new β-cells has the potential to be a real solution, but current methods struggle to produce cells that are both fully functional and safe. At Karolinska Institutet's ATMP Center for Cell and Gene Therapy, our two research groups are collaborating to develop the next generation of β-cells using synthetic biology. One of our research groups has recently developed a very promising new method to develop β-cells from stem cells, but unfortunately this method still gives rise to immature and unwanted cell types. To solve just such problems, the second of our groups has developed PreciCE, a method integrating advanced gene control and machine learning algorithms to control cell identity with high precision. PreciCE can control cell development more precisely by simultaneously promoting the right genes and suppressing the wrong ones. The goal of our research is to create mature and safe β-cells on a large scale for preclinical and clinical testing. If successful, this project could pave the way for cost-effective and widespread access to β-cell therapy - an important step towards a cure for type 1 diabetes.