2019 Team

miR-Based Therapy

This proposal aims at the generation of insulin producing beta-cells using miRNA-modified pluripotent cells (PSCs) as a new therapy to treat Type I diabetes, an autoimmune disease that destroys the β cells leading to glucose metabolic failure.

In 2018, around 2.8 Mio people were diagnosed with Type I diabetes. Currently, cell-based therapies including endocrine islet transplantation raises hopes towards developing a cure for diabetes. However, due to immune-rejection and time-limited functionality, the transplantation of donor-pancreatic islets has been almost dismissed.

In turn, the stem cell-based therapy has emerged as an encouraging alternative. Nevertheless, the complications still arise from poor quality of PSCs and lack of differentiation capacity and functionality of the developed tissue.

Our proposal would overcome these limitations thereby efficiently generating functional β cells.

We have found that transient exposure to one miRNA significantly expands the differentiation capacity of pluripotent and multipotent cells. Interestingly, these modified cells are capable of producing pancreas in teratoma assays in vivo and induce the production of insulin in pancreatic organoids in vitro.

Exposure of PSCs to this miRNA might enhance not only the generation of insulin-producing pancreatic β cells but also its functionality, thus improving significantly current procedures to generate either general or patient-specific IPCs for treating diabetes. The results of this project can significantly help these patients and their quality of life, improve the management of the disease, avoid secondary effects and diseases derived from diabetes, etc. The positive impact of this technology would be therefore beyond diabetes per se.

Team Lead 
Maria Salazar-Roa

Spanish National Cancer Research Center (CNIO)

MIT Institute for Medical Engineering and Science (IMES)

Lead Mentors
Petra Krauledat
Peter Hansen