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Small molecules plus transient expression of transcription factors to convert fibroblasts to pancreatic b cells

Fibroblasts transiently expressing transcription factors and treated with small molecules could provide transplantable pancreatic b cells to help treat type 1 diabetes. In mouse fibroblast cultures, transient expression of four pluripotency transcription factors plus delivery of specific small molecule combinations produced transdifferentiated endoderm-like cells that could be further converted into pancreatic progenitor-like cells and all three pancreatic lineages. In a mouse model of hyperglycemia, transplanted pancreatic progenitor-like cells gave rise to all three pancreatic cell types, including glucose-responsive, insulin-producing b cells, which reduced hyperglycemia. Next steps include translating the approach to human cells and trying to substitute the genetic introduction of pluripotency factors for noninvasive methods.

SciBX 7(9); doi:10.1038/scibx.2014.272
Published online March 6, 2014

Patent application filed; available for licensing

Li, K. et al. Cell Stem Cell; published online Feb. 6, 2014;
Contact: Sheng Ding, University of California, San Francisco, Calif.